Search results from the Australian New Zealand Clinical Trials Registry

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Keyword: cannabidiol
Recruitment Status: Recruiting

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A Randomised, Double-Blind, Vehicle-Controlled Study of the Safety and Tolerability of BTX 1702 in Patients with Papulopustular Rosacea

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Trial Information

Trial summary

Botanix is developing BTX 1702 for the topical treatment of moderate to severe papulopustular rosacea.
This is a randomised, double-blind, vehicle-controlled, parallel-group, Phase 1b study in adult patients, aged 18-65 with moderate or severe papulopustular rosacea. The objective of this study is to determine the safety and tolerability of BTX 1702 10% (w/w) Gel and BTX 1702 20% (w/w) Gel in patients with papulopustular rosacea.
Eligible patients will be enrolled and randomised 1:1:1 to BID treatment with BTX 1702 10% (w/w) Gel, BTX 1702 20% (w/w) Gel, or Vehicle Gel for a planned 56 days. Approximately one hundred and twenty (120) patients (80 active: 40 vehicle) will be enrolled.
The primary outcome for the study is to determine the safety and tolerability of BTX 1702 10% (w/w) Gel, BTX 1702 20% (w/w) Gel, or BTX 1702 Vehicle Gel following 56 days BID applications in patients with papulopustular rosacea.
The safety and tolerability outcome measures will be assessed through the collection and review of AE's, laboratory parameters evaluated throughout the duration of the trial and via information recorded in participant diaries.
Exploratory analysis will be conducted via change in inflammatory lesion counts, change in Investigator’s Global Assessment (IGA-PP), change in Clinician’s Erythema Assessment (CEA) scale and Patient Reported Outcomes (PRO).

Broad Health ConditionPapulopustular Rosacea

Specific Health ConditionSkin
Dermatological conditions

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,WA,VIC

Trial location outside Australia

Country
New Zealand

Anticipated date of first participant enrolment21/06/2021

Anticipated date of last participant enrolment10/05/2022

Phase of TrialPhase 1

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

#1. Patient has a diagnosis at Screening and Baseline of moderate or severe papulopustular rosacea of the face with:
a. 15 to 75 (inclusive) inflammatory lesions (papules/pustules) on the face.
b. Rosacea severity of moderate (3) or severe (4) on a 5-point static investigator global
assessment for papules & pustules (IGA-PP)
c. CEA score of 3 or 4 (moderate or severe) assessed on the face.
d. No more than 5 large open comedones.

#2. An independent reviewer will review screening photographs to confirm eligibility of the patient for enrolment. No patient may be randomized prior to the confirmation of eligibility by the independent reviewer.

#3. Patient has less than or equal to 2 nodular lesions (greater than or equal to 5 mm in diameter).

#4. Patient agrees to abstain from use of marijuana or cannabidiol (CBD) products throughout the study.

#5. Male patients and their partners must agree and commit to use a barrier method of
contraception throughout the study and for 90 days after last study medication application.

Minimum age18 Years

Maximum age65 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

#1. Patient has used any marijuana products, via any route, within 4 weeks prior to the
Screening Visit. A positive urine drug screen (UDS) for tetrahydrocannabinol (THC) will
exclude the patient. Patient may be deemed eligible if the UDS identifies patient-reported, prescribed medications or appropriate levels of alcohol, as determined by the investigator.

#2. Patient has any significant active infection at Baseline.

#3. Patient has known human immunodeficiency viruses (HIV) infection or hepatitis B or C.

#4. Patient has initiated a hormonal method contraception within 3 months of Baseline or plans to discontinue or change during the study or changed product within 3 months of Baseline.

#5. Patient has used topical acne or rosacea treatments, including metronidazole, azaleic acid, sulfacetamide, salicylic acid, benzyl peroxide, ivermectin, bromonidine, or oxymetazoline within 4 weeks of Baseline.

#6. Patient has used systemic retinoids or high dose (less than 10,000 IU/day) Vitamin A, within 90 days of Baseline.

#7. Patient has used topical or systemic antibiotics within 4 weeks of Baseline.

#8. Patient has used topical (facial) or systemic anti-inflammatories for more than 14
consecutive days in the 4 weeks prior to Baseline.

#9. Patient has used topical (facial) or systemic corticosteroids 4 weeks prior to Baseline.

#10. Patient has used vasodilating agents (e.g., anti-hypertensives, erectile dysfunction
medications, nitroglycerin) 6 weeks prior to Baseline.

#11. Patient has used alpha-adrenergic receptor-blocking agents 6 weeks or alpha-adrenergic agonists 4 weeks prior to Baseline.

#12. Patient has ocular rosacea and/or blepharitis/meibomianitis and requires treatment by an ophthalmologist during the study.

#13. Patient has sunburns, unevenness in skin tones, tattoos, scars, excessive hair (e.g., beard, moustache), freckles, birthmarks, moles, oedematous lesions or other skin damage or abnormality that would result in the inability to perform study assessments.

#14. Patient has an active or potentially recurring skin conditions(s) other than rosacea that may interfere with the rosacea assessment of or require topical or systemic treatment that may affect treatment assessments.

#15. Patient has used systemic or other immunosuppressive medications within 4 weeks of the Baseline Visit (inhaled corticosteroid less than or equal to 1000 µg daily dose is acceptable).

#16. Patient has used phototherapy 14 days prior to Baseline or has had excessive sun exposure with intent to sunbathe or tan or use artificial tanning agents.

#17. Patient has undergone dermatologic procedures to the face including laser, intense pulsed light, chemical peels, salabrasion, dermabrasion or botulinum toxin injection within 4 weeks of the Baseline Visit.

#18. Patient has other dermatological conditions that require the use of interfering topical or systemic therapy or that might interfere with study assessments such as, but not limited to, acne or atopic dermatitis.

#19. Patient has participated in another investigational medication or device research study within 30 days of the Screening Visit or five half-lives of the medication, whichever is longer.
Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Botanix Pharmaceuticals Ltd
Primary Sponsor Address: Level 1, 50 Angove Street, North Perth, Western Australia 6005
Primary Sponsor Country: Australia

Trial IDACTRN12621000689875

Contact person for information and recruitmentMr
Matt Callahan
Botanix Pharmacueticals, 3602 Horizon Drive, Suite 160, King of Prussia, PA 19406
+1 445 300 3403

Further information iconmcallahan@botanixpharma.com
United States of America

Cannabidiol (CBD) treatment for insomnia

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Trial Information

Trial summary

Insomnia is a highly prevalent and burdensome disease. It is thought to affect as many as one in three adults worldwide at any one time. Treatment options for insomnia are often limited to doctor-implemented cognitive behavioural training (CBT), pharmaceutical intervention, or a combination of the two. Whilst traditionally effective, both options have considerable limitations, most frequently those associated with cost, compliance and effectiveness (CBT), or tolerance and addiction potential (pharmaceutical). 

The Australian Federal Government recently approved the medical use of cannabinoids among select patient groups. The main active ingredient, cannabidiol (CBD), may have sedating and/or sleep-enhancing effects. There is therefore increasing global interest as its potential use as an alternate sleep-aid.

The primary objective of this research is to examine whether 2 weeks of nightly supplementation with a novel sublingual CBD-treatment improves sleep outcomes among individuals with moderate-severe insomnia. Specifically, we seek to examine the effect of treatment on subjective [sleep onset latency, sleep efficiency (%), and time awake after sleep onset] and objective sleep quality [as measured by Actigraphy outcomes (time in bed, sleep efficiency (%), number of awakenings, sleep latency) after 2-week supplementation with sublingual CBD compared with a placebo.

Broad Health Conditioninsomnia

Specific Health ConditionNeurological
Other neurological disorders

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
VIC

Anticipated date of first participant enrolment2/03/2020

Anticipated date of last participant enrolment1/06/2020

Phase of TrialPhase 1 / Phase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Males and Females Aged 18-45 years
2.	Insomnia Severity Index (ISI) score of 15 or more.
3.	Self-report difficulties falling and / or staying asleep (yes)
4.	Dissatisfaction with current sleep patterns (yes)
5.	Sleep problems interfering with daily life (yes)

Minimum age18 Years

Maximum age45 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1. Inability to speak or read English
2. Current shift worker
3. History of severe neurological, cardiac, endocrine, gastrointestinal, or bleeding disorders (self-report)
4. History of major psychiatric disorder in the past 12 months except clinically-managed mild depression
5. Severe current depression (BDI score of greater than or equal to 20)
6. Severe current anxiety (BAI score greater than or equal to 16)
7. Pregnancy or lactation - males and females shall be advised to use reliable contraception for the duration of drug therapy and a urine pregnancy test will be performed for female participants where necessary.
8. Use of any CNS-active drugs (including antidepressants, opioids, benzodiazepines) for the past 3 months or at the medical officer’s discretion
9. Excessive caffeine use that in the opinion of the investigator contributes to the participant's insomnia
10. Other pre-existing sleep disorder (restless legs syndrome, narcolepsy, parasomnias etc.) (self-report)
11. Used any modality of treatment for insomnia, including CBT, within 3 months before screening or at the medical doctor's discretion
12. History of drug or alcohol dependency or abuse within approximately the past 2 years (self-report and at medical officer’s discretion)
13. Probable moderate to severe obstructive sleep apnoea (STOP-BANG questionnaire)
14. Currently participating in any other trials involving investigational or marketed products within 30 days prior to the screening visit.
15. Not willing to abstain from driving, riding, operating heavy machinery for 8 hours after taking the nightly treatment. 

The following interim exclusion criteria will occur on conclusion of the one-week placebo-run in period and prior to randomisation into the treatment arms (CBD vs placebo).
1. Placebo responder. Placebo responders will be identified and eliminated from the trial before randomisation. A placebo responder will be defined as any person entering the trial who significantly changes their subjective sleep scores derived from the sleep diary during the 7-day placebo run-in. This will be operationally defined as an individual whose:
o Subjective Sleep Efficiency (SE) goes above 85%
o Subjective Sleep Onset Latency (SOL) goes below 31 minutes
o Subjective Wake after Sleep Onset (WASO) goes below 31 minutes, as determined by the Subjective sleep information questions.

2. Non-compliers. Non-compliers will be identified as those;
a. Missing greater than or equal to 20% of the treatment doses
b. Using excessive amounts of the treatment (placebo) (greater than 20ml used).
c. Missing greater than or equal to 20% days wearing the GENEActiv wrist-mounted activity monitor over the one-week run-in period (if assigned).
Contact details and further information

Sponsor Primary Sponsor Type: University
Primary Sponsor Name: Swinburne University of Technology
Primary Sponsor Address: John St, Hawthorn VIC 3122
Primary Sponsor Country: Australia

Trial IDACTRN12620000070932

UTNU1111-1246-0810

Contact person for information and recruitmentDr
Amie Hayley
Swinburne University of Technology, Faculty of Health, Arts and Design School of Health Sciences Centre for Human Psychopharmacology Mail H24 PO Box 218 Hawthorn, Victoria, 3122
+61 3 92145585

Further information iconahayley@swin.edu.au
Australia

Clinical Study of Synthetic Cannabidiol in Children and Adolescents with 22q11.2 Deletion Syndrome

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Trial Information

Trial summary

This is an open-label single-center study, to assess the safety, tolerability and efficacy of cannabidiol administered as ZYN002, a transdermal gel, for the treatment of child and adolescent patients with 22q11.2 Deletion Syndrome (22qDS). Male and female patients with 22qDS will be treated for 14 weeks. Patients taking Anti Epileptic Drug medications will have an additional one or two week Taper Period after the completion of doing with ZYN002. Approximately 20 male and female patients, ages 6 to < 18 years, will receive ZYN002.

Broad Health ConditionPediatric 22q11.2 deletion syndrome

Specific Health ConditionNeurological
Other neurological disorders
Mental Health
Psychosis and personality disorders
Human Genetics and Inherited Disorders
Other human genetics and inherited disorders

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
QLD

Hospital
Queensland Children's Hospital - South Brisbane

Postcode
4101 - South Brisbane

Anticipated date of first participant enrolment3/06/2019

Anticipated date of last participant enrolment26/02/2021

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Male or female children and adolescents aged 6 to <18 years, at the time of Screening.
2.	Judged by the Investigator to be in generally good health at Screening based upon the results of a medical history, physical examination, and clinical laboratory test results.  Laboratory results outside of the reference range must be documented as not clinically significant by the Investigator.  
3.	Patients must have a diagnosis of 22qDS confirmed by genetic testing, with or without autistic features.
4.	Patients have a CGI-S score of 4 or higher at Screening and Visit 2.
5.	Patients must have a score on the ABC-C Irritability Subscale of 18 or higher at Screening and Visit 2.
6.	Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of one or two AEDs, or must be seizure-free for one year if not currently receiving AEDs.
7.	If patients are receiving non-pharmacological behavioral and/or dietary interventions, they must be stable for three months prior to Screening.
8.	Patient has demonstrated stable calcium levels for one year prior to Screening.
9.	Patients have a body mass index between 12–30 kg / m2 (inclusive).
10.	Females of childbearing potential must have a negative pregnancy test at the Screening Visit and a negative pregnancy test at all designated study visits.
11.	Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
12.	Patients and parents/caregivers must be adequately informed of the nature and risks of the study and give written informed consent (and assent if applicable) prior to Screening.
13.	Parents/caregiver(s) must provide written consent to assist in study drug administration.
14.	In the Investigator’s opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.

Minimum age6 Years

Maximum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1.	Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication.
a.	Standard acceptable methods of contraception include abstinence or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, vasectomy, or intrauterine device.
2.	History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
3.	Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study.
4.	Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels less than or equal to 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels less than or equal to 3 times the ULN as determined from Screening safety laboratories.
5.	Use of cannabis or any THC or CBD-containing product within three months of Screening Visit or during the study.
6.	Patient has a positive drug screen for sympathomimetic amines (amphetamines (unless prescribed); benzodiazepines; buprenorphine; cannabinoids; methadone; cocaine (metabolites); and opiates; (excludes midazolam used at blood draws, and barbiturates used as AED medication), including ethanol.
7.	Patient is using the following AEDs: phenobarbital, ethosuximide, felbamate, or vigabatrin.
8.	Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including but not limited to the following medications: midazolam (except single doses administered for the purposes of obtaining blood samples and ECG’s), oral ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin, vincristine, vinorelbine, and St. John’s Wort.
9.	Patient with diagnosis of known genetic disorder, other than 22qDS (i.e. Prader-Willi Syndrome, Angelman Syndrome, Fragile X Syndrome, Rett Syndrome etc.).
10.	Patient has diagnosis of DiGeorge or Velocardiofacial syndrome without the presence of 22qDS.
11.	Patient has a primary psychiatric diagnosis other than 22qDS or anxiety, including bipolar disorder, psychosis, schizophrenia, post-traumatic stress disorder (PTSD) or major depressive disorder. 
12.	Patients is on stable treatment of >6 months of not more than two psychoactive medications at screening or throughout the study (with the exception of one psychoactive medication prescribed for sleep). 
13.	Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
14.	Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
15.	Patient has an acute or progressive neurological disease, or any psychiatric disorder or severe mental abnormalities that are likely to require changes in drug therapy or interfere with the objectives of the study or ability to adhere to protocol requirements.
16.	Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.
17.	Patients at risk of needing cardiovascular surgical repair within the upcoming 12 months.
18.	Patient has unstable cardiovascular disease, such as advanced arteriosclerosis, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), other serious or other clinically unstable  cardiac problems.
19.	Clinically unstable cardiovascular disease as indicated by history, physical examination or ECG. 
20.	Presence of a moderate or severe surgically uncorrected structural cardiac abnormality.
21.	History of major congenital  heart disease including those who had surgical repair.  Congenital heart disease is defined by classification of complex (fontan circulation, hypoplastic left heart, double inlet ventricle, double outlet right ventricle, Eisenmenger’s syndrome, mitral atresia, tricuspid atresia, pulmonary atresia (with or without intact ventricular septum) , congenitally corrected transposition of great arteries, complete atrioventricular septal defect, truncus arteriosus, other single ventricle physiology, other cyanotic congenital heart disease), moderate (transposition of great vessels, tetralogy of fallot, total or partial anomalous pulmonary venous drainage, Ebstein’s anomaly, coarctation of aorta, aortic stenosis, pulmonary stenosis, ostium primum atrial septal defect, sinus of valsalva fistula/aneurysm) or simple (ventricular septal defect, atrial septal defect, persistent ductus arteriosus, mild pulmonary stenosis).
22.	Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication.
23.	Any skin disease or condition, including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration, that may affect treatment application, application site assessments, or absorption of the study drug.
24.	History of treatment for, or evidence of, drug abuse within the past year.
25.	Patient responds “yes” to Question ‘4’ or ‘5’ on the C-SSRS (Children) during Screening or at any time on study.
Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals Pty Ltd
Primary Sponsor Address: 2 Riverside Quay Southbank, VIC 3006
Primary Sponsor Country: Australia

Trial IDACTRN12619000673145

Contact person for information and recruitmentDr
Nancy R Tich
Zynerba Pharmaceuticals Inc., 80 West Lancaster Ave., Suite 300, Devon, PA 19333
+1-973-727-4117

Further information icontichn@zynerba.com
United States of America

An open label, study to evaluate the safety of Cannabidiol (CBD) for the prevention of Acute Graft-Versus-Host Disease (GVHD) after allogeneic hematopoietic cell transplantation.

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Trial Information

Trial summary

The purpose of this study is to evaluate the safety of cannabidiol (CBD) for the prevention of Acute Graft-Versus-Host Disease (GVHD) in patients who are undergoing allogeneic hematopoietic cell transplantation.

Who is it for?
You may be eligible for this study if you are aged 18 or older, and are undergoing a allogeneic hematopoietic stem cell transplantation (HSCT).
Study details
Participants in this study will consume a solution of cannabidiol (CBD) in olive oil by mouth twice per day from the time transplant procedures commence until 98 days post-transplant. The concentration of CBD will vary depending on when you enrol in the study. As part of his study, all participants will have blood and urine tests, and undergo physical examinations, in addition to standard post-transplant tests.

It is hoped this research might provide some evidence that CBD can prevent acute Graft Versus Host Disease (GVHD) in patients that have undergone an allogeneic HSCT, including how it is tolerated, absorbed and interacts within the body.

Broad Health ConditionAcute Graft Versus Host Disease
Hematopoietic Stem Cell Transplantation

Specific Health ConditionCancer
Other cancer types
Blood
Haematological diseases

Trial FocusPrevention

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW

Hospital
St Vincent's Hospital (Darlinghurst) - Darlinghurst

Postcode
2010 - Darlinghurst

Trial location outside Australia

Country
Israel

Anticipated date of last participant enrolment30/04/2019

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Any malignant hematological disease in CR or Myelodysplastic Syndrome (MDS)
2.	Age greater than or equal to 18 years 
3.	Karnofsky Score (KS) greater than or equal to 60% 
4.	HSCT-Comorbidity Index (HSCT-CI) score less than or equal to 3
5.	No major organ dysfunction
6.	Myeloablative or reduced intensity conditioning regimen
7.	matched (7/8 or 8/8) unrelated donor 
8.	Peripheral blood stem cell graft
9.	Subject’s written informed consent

Minimum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1.	Malignant hematological disease, other than MDS, not in CR
2.	Myelofibrosis
3.	Allogeneic transplantation from a matched or mismatched sibling donor 
4.	Cord blood transplantation
5.	Positive serology for HIV
6.	Serious psychiatric or psychological disorders
7.	Any uncontrolled infection at time of registration
8.   Study subjects must not be pregnant or breastfeeding or planning to become pregnant or breastfeed during the course of the trial, as well as within 30 days after the anticipated date the subjects would complete the study. 
9.	Active consumption of illicit drugs (such as: Crack cocaine, Heroin, Methamphetamines, Cocaine, Bath Salts, Amphetamines, Methadone, Benzodiazepine, Marijuana, Ecstasy)
10. Use of Cannabis and/or its derivatives fourteen days prior to HSCT and for the duration of study participation 
11. QTc greater than 450ms per Friderica's correction and impaired cardiac  function or clinically significant cardiac disease
12. Inadequate renal function defined as measured creatinine clearance greater than 2.0 mg/dl
13. Liver enzymes: ALT and AST greater than 3 times the upper limit of normal
14. Pregnant or breastfeeding (positive serum beta-HCG 7 days before first dose)
15. Treatment with another investigational drug, biological agent, or device within 30 days of first dose, or investigational cell therapy within 6 months of first dose..

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Kalytera Therapeutics Israel Ltd
Primary Sponsor Address: Technology Building, Road #4 Katzrin 1290005, Israel
Primary Sponsor Country: Israel

Trial IDACTRN12619000623190

UTNU1111-1229-9861

Contact person for information and recruitmentMs
Toula Papadodimitrakis
Salzman Group CRO 270 Ferntree Gully Road Notting Hill Vic 3168
+613 8375 7156 ext 2601

Further information icontoula@salzgrp.com
Australia

A study to evaluate the effect of food on the pharmakokinetics and cardiac function in male and female healthy volunteers who have been administered cannibidiol.

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Trial Information

Trial summary

This research project is being conducted to investigate the safety and tolerability of multiple doses of cannabidiol (CBD) when administered to healthy volunteers.

Broad Health ConditionAcute Graft Versus Host Disease

Specific Health ConditionInflammatory and Immune System
Other inflammatory or immune system disorders

Trial FocusPrevention

Recruitment statusRecruiting

Recruitment Details
Recruitment State
VIC

Hospital
Nucleus Network - Melbourne

Postcode
3004 - Melbourne

Anticipated date of last participant enrolment24/05/2019

Phase of TrialPhase 1

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Healthy adult subjects =18 – 60 years of age
2.	Body Mass Index (BMI) of 18 to 30 kg/m2 inclusive and a minimum weight of at least 50 kg at screening and not more than 100 kg at screening
3.	Non-users of tobacco or nicotine-containing products (minimum of 4 weeks from admission)
4.	Subjects with reproductive potential required to practice abstinence or be using and willing to continue using a medically acceptable form of birth control for at least one month prior to screening (at least 3 months for oral contraceptives) and for at least 60 days after the last study drug administration.
5.	Free from any clinically significant abnormality based on medical history, vital signs, physical examination, ECG, and laboratory evaluation at screening and admission to the treatment session, as judged by the investigator or designee.
6.	Systolic blood pressure between 90 to 140 mmHg and diastolic blood pressure between 50 and 90 mmHg at screening and admission to the treatment session.
7.	Male Participants who agree to the following during their participation in this study and for at least 1 month after the last dose of study intervention: Refrain from donating sperm PLUS either: 
o	Be abstinent from heterosexual [or homosexual] intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent OR Must agree to use contraception/barrier as detailed below: 
o	Agree to use a male condom (and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak) when having sexual intercourse with a woman of childbearing potential who is not currently pregnant
8.	A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
o	Is not a woman of childbearing potential (WOCBP) OR
o	Is able to abstain from heterosexual intercourse if it is in keeping with her preferred and usual lifestyle (such as participants with same-sex partners)
OR
o	Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of <1% per year), during the intervention period and for at least 30 days plus 30 days (a menstrual cycle) after the last dose of study intervention [and agrees not to donate eggs (ova, oocytes) for the purpose of reproduction during this period.]. The investigator should evaluate the effectiveness of the contraceptive method in relationship to the first dose of study intervention.
o	A WOCBP must have a negative pregnancy test [ serum] as required by local regulations) within [24 hours] before the first dose of study intervention.
9.	Subjects must be able to speak, read and understand English sufficiently to understand the nature of the study and provide written informed consent
10.	Subjects must be capable of giving signed informed consent and agree to comply with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
11.	Subjects must have understood and provided written informed consent prior to initiation of any protocol-specific procedures.

Minimum age18 Years

Maximum age60 Years

GenderBoth males and females

Can Healthy volunteers participate?Yes

Key exclusion criteria

1.	History of clinically significant cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, and renal or other major disease as determined by the Investigator and/or Sponsor’s Medical Monitor
2.	Evidence of disease that may influence the outcome of the study within 4 weeks before dosing as determined by the Investigator and/or Sponsor’s Medical Monitor
3.	Any history of gastrointestinal surgery that may affect the pharmacokinetic profile of the study drug
4.	Any clinically abnormal symptom or organ impairment found by medical history, physical examinations, vital signs, ECG findings or laboratory test results that requires medical treatment at Screening or Baseline
5.	History of any medical condition which, in the opinion of the Investigator, may interfere with study procedures or compromise subject safety (e.g. psychiatric illness, disability or social situation)
6.	Known history of clinically significant drug allergy (including to study drugs or any of their excipients) at Screening or Baseline
7.	Known history of clinically significant food allergies or presently experiencing significant seasonal or perennial allergy at Screening or Baseline
8.	Active viral hepatitis (B or C) and HIV as demonstrated by positive serology at Screening 
9.	Current or chronic history of liver disease, known hepatic or biliary abnormalities (with the exception of Gilbert’s syndrome or asymptomatic gallstones) 
10.	Alanine transaminase (ALT) >1.5x upper limit of normal (ULD)
11.	Bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if fractionated and direct bilirubin <35%)
12.	Persistent systolic blood pressure (BP) > 140 mmHg or < 90 mmHg and diastolic BP >90mmHg or <40mmHg at Screening or Baseline
13.	Heart rate <50 or >100 beats/minute at Screening
14.	History of prolonged QT/QTc interval
15.	A history of risk factors for torsade de pointes or the use of concomitant medications that prolong the QT/QTc interval
16.	Corrected QT interval (QTc) >450 milliseconds (msec) (for males) or >470 msec (for females)
•	The QTc is the QT interval corrected for heart rate according to Bazett’s formula (QTcB), Fridericia’s formula (QTcF), and/or another method, machine-read or manually over-read
•	The specific formula that will be used to determine eligibility and discontinuation for an individual participant should be determined prior to initiation of the study. In other words, several different formulae cannot be used to calculate the QTc for an individual participant and then the lowest QTc value used to include or discontinue the participant from the trial.
•	For purposes of data analysis, QTcB, QTcF, another QT correction formula, or a composite of available values of QTc will be used 
17.	A history of additional risk factors for TdP (e.g. heart failure, hypokalemia, family history of Long QT Syndrome)
18.	History of myocardial infarction or active ischemic heart disease
19.	History of clinically significant arrhythmia or uncontrolled arrhythmia
20.	Intake of caffeinated beverages or food within 72 hours before dosing
21.	Intake of nutritional supplements, juice, and herbal preparations or other foods or beverages that may affect the various drug metabolizing enzymes and transporters (e.g. grapefruit, grapefruit juice, apple or orange juice, vegetables from the mustard green family) within 1 week before dosing. Alcohol within 72 hours of dosing.
22.	Intake of herbal preparations containing St. John’s Wort within 4 weeks before dosing
23.	Past or intended use of over-the-counter (OTC) or prescription medications 2 weeks before first dosing unless agreed by the Investigator and Sponsor’s Medical Monitor to not be clinically relevant
24.	Live vaccine(s) within 1 month prior to screening, or plans to receive such vaccines during the study 
25.	Regular alcohol consumption within 3 months prior to the study defined as: 
•	An average weekly intake of >14 units for males and females. One unit is equivalent to 8g of alcohol: pot of beer (~240mL), 1 glass (125mL) of wine or 1 (25mL) measure of spirits
26.	History of drug or alcohol dependency or abuse within the 2 years before Screening, or who have a positive urine drug or alcohol test at Screening or Baseline
27.	Engagement in strenuous exercise within 72 hours before dosing 
28.	Subject is a female who is pregnant, nursing, or breastfeeding, or who intends to become pregnant during the participation in the study. 
29.	Currently enrolled in another clinical trial or used any investigational drug or device within 30 days preceding informed consent 
30.	A subject who, in the opinion of the investigator or designee, is not considered to be suitable and is unlikely to comply with the study protocol for any reason.
Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Kalytera Australia Pty Ltd
Primary Sponsor Address: Level 4, 68 Georges Terrace Perth, WA 6000 Australia
Primary Sponsor Country: Australia

Trial IDACTRN12619000443190

UTNKAL07

Contact person for information and recruitmentMs
Antu Mishra
Nucleus Network 5th Floor Burnet Tower AMREP Precinct 89 Commercial Road Melbourne 3004 VIC
+61 3 8593 9259

Further information icona.mishra@nucleusnetwork.com.au
Australia

Cannabinoids for Symptom Control in Advanced Cancer, an Open Label Prospective Clinical Trial in New South Wales (NSW)

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Trial Information

Trial summary

The main purpose of this study is to determine what dose and frequency of medicinal cannabis is the best to relieve key symptoms in people with advanced cancer.

Who is it for?
You may be eligible to join this study if you are aged 18 years or more, have a confirmed diagnosis of advanced cancer, with significant symptoms of severe pain, and/or vomiting, and/or nausea and/or lack of appetite that are poorly controlled by usual treatments, and a predicted life expectancy of more than 3 months and less than 12 months.

Study details
All participants in the study will be prescribed one of a number of cannabis medicines. The cannabis product will be given in addition to the usual treatments for their advanced cancer symptoms. On the first day of treatment participants will have 5 blood samples collected over 4 hours to measure the levels of cannabis medicines in their blood. They will also complete some online questionnaires about how they feel and be monitored for side effects.

Participants will remain enrolled in this trial and receive the cannabis medication prescribed at no cost until they are no longer able to take it or withdraw from the trial. Participants will be asked to complete regular online questionnaires and have a single blood sample taken every few weeks when they see the trial doctor, until they no longer receive the product and/or withdraw from the trial.

It is hoped the information collected in this study will guide the use of cannabis medicines to control symptoms in people with advanced cancer.

Broad Health ConditionCancer
Pain
Anorexia
Nausea

Specific Health ConditionCancer
Any cancer
Anaesthesiology
Pain management

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW

Hospital
Orange Health Service - Orange

Hospital
The Chris O’Brien Lifehouse - Camperdown

Hospital
Liverpool Hospital - Liverpool

Hospital
Westmead Hospital - Westmead

Hospital
Prince of Wales Hospital - Randwick

Hospital
Gosford Hospital - Gosford

Hospital
Wyong Public Hospital - Hamlyn Terrace

Hospital
Tamworth Rural Referral Hospital - Tamworth

Hospital
St Vincent's Hospital (Darlinghurst) - Darlinghurst

Hospital
Wollongong Hospital - Wollongong

Hospital
Shoalhaven Hospital - Nowra

Hospital
Macquarie University - North Ryde

Postcode
2800 - Orange

Postcode
2050 - Camperdown

Postcode
2170 - Liverpool

Postcode
2145 - Westmead

Postcode
2031 - Randwick

Postcode
2250 - Gosford

Postcode
2259 - Hamlyn Terrace

Postcode
2340 - Tamworth

Postcode
2010 - Darlinghurst

Postcode
2500 - Wollongong

Postcode
2541 - Nowra

Postcode
2109 - North Ryde

Anticipated date of first participant enrolment25/02/2020

Anticipated date of last participant enrolment1/02/2023

Phase of TrialPhase 4

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1. Aged 18 years or older.
2. Has provided written Informed Consent for participation in this trial.
3. Confirmed advanced cancer (any) with or without stable disease.
4. Predicted life expectancy of 3 to 12 months at time of consent, as estimated by the study investigator and confirmed by an additional qualified clinician, independent of the trial.
5. Persistent symptoms of anorexia, nausea or refractory pain, which in the investigator’s opinion are not responsive to implementation of standard practice, and who are ineligible or not wishing to be involved in other clinical trials of cannabis medicines.
6. Willing and able, in investigator’s opinion, to comply with all trial requirements, including keeping a patient diary, completing clinical measurement scales and having pharmacokinetic samples collected as per Participant Information Sheet. Note: completion of these trial requirements is essential and intentional non-compliance may cease access to trial product and will be managed on a case-by-case basis. Non-compliance due to declining health is unintentional and it is hoped unintentional noncompliance due to declining health may be minimised through optional proxy completion of patient reported outcomes and patient diary.
7. Available to attend Trial Site for follow up and collection of investigational product.
8. Participants (and/or partners) capable of childbearing are using adequate contraception.

Participants are able to continue to receive standard concomitant care and interventions, such as chemotherapy and radiotherapy, and take medications for other conditions as usual in the advanced care population

Minimum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1. Lack of capacity to consent.
2. Unwilling to keep a Patient Diary, attend follow up appointments, complete clinical measurement scales or have pharmacokinetic samples. 
3. History of schizophrenia, other psychotic illness, severe borderline personality disorder, or other significant psychiatric disorder other than depression or anxiety associated with underlying condition. Delirium is an excluded condition.
4. Previous severe adverse event to any cannabinoid product, such as cannabis related psychosis, panic attack, palpitations.
5. A diagnosed substance use disorder (ICD-10 criteria (abuse, dependence)) to alcohol, opioids, cannabis, benzodiazepines, or illicit stimulants. Nicotine and caffeine are excluded. 
6. Known allergy to cannabis or other ingredient of cannabis medicine e.g. carrier oil.

Note: Prior access to authorised cannabis medicines or current use of illicit cannabis is not an exclusion, unless associated with a serious adverse event (SAE) or substance use disorder as per exclusion criteria above.
Contact details and further information

Sponsor Primary Sponsor Type: Government body
Primary Sponsor Name: Health Administration Corporation acting through the NSW Ministry of Health
Primary Sponsor Address: Medical Advisor Office of the Chief Health Officer, NSW Health 73 Miller Street North Sydney NSW 2060
Primary Sponsor Country: Australia

Trial IDACTRN12619000265178

UTNU1111-1227-1657

Contact person for information and recruitmentDr
Rachel Galettis
Clinical Trial Coordinator ACRE C/- Hunter Medical Research Institute Lot 1, Kookaburra Circuit, New Lambton Heights, NSW, 2305
+61 2 40420915

Further information iconACRE-CARE@newcastle.edu.au
Australia

A study that evaluates the effectiveness of oral combined THC/CBD for people with advanced cancer experiencing a range of symptoms.

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Trial Information

Trial summary

The purpose of this study is to assess whether delta-9-tetrahydrocannabinol (THC) and cannabidoil (CBD) can be used to reduce total symptoms in patients with advanced cancer in palliative care.

Who is it for?

You may be eligible for this study if you are over 25 years of age and have been diagnosed with advanced cancer.

Study details

Participants will be randomly assigned to one of two treatment groups; either THC/CBD or a placebo medication. 

Participants will be asked to take increasing doses of the study medication for 14 days, with the dose increasing until participants are satisfied with the symptom improvement and are experiencing no unacceptable side effects. After these 14 days, participants will be asked to take a steady dose of the medication for another set of 14 days. 

During the 28 days of the study you will be required to have routine bloods and urine test which will be used as part of the eligibility and post trial analysis 

It is hoped that this research will show a positive effect of THC/CBD on symptoms for patients suffering with advanced cancer and thus provide an option in helping manage symptoms.

Broad Health ConditionCancer
Fatigue
Nausea
Breathlessness
Appetite
Psychological effects

Specific Health ConditionCancer
Any cancer

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
QLD

Hospital
Mater Adult Hospital - South Brisbane

Hospital
Mater Private Hospital - South Brisbane

Hospital
St Vincent's Hospital Brisbane - Kangaroo Point

Hospital
Royal Brisbane & Womens Hospital - Herston

Hospital
Princess Alexandra Hospital - Woolloongabba

Hospital
Gold Coast Hospital - Southport

Postcode
4101 - South Brisbane

Postcode
4169 - Kangaroo Point

Postcode
4029 - Herston

Postcode
4102 - Woolloongabba

Postcode
4215 - Southport

Anticipated date of first participant enrolment1/04/2019

Anticipated date of last participant enrolment1/04/2022

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

Patients with advanced histologically proven cancer (metastatic or locally advanced) known to the palliative care team of the recruiting centre who:

- have an ESAS TSDS greater than 10 for cancer-related symptoms*
- at least one individual ESAS score greater than 3
- AKPS score >30
- aged >25yrs and above. English speaking (or have interpreter available)
- have a negative pregnancy urine test at eligibility (only if of reproductive potential) and 
  agree to avoid pregnancy during the study and 12 weeks following the last dose of the 
  study drug. Males must agree to avoid fathering a child and to not donate sperm during 
  the study and for at least 12 weeks following the last dose of the study drug
- have a negative THC urine test
- able to tolerate oral medication 
- willing to receive standard palliative care
- comply with trial requirements; agree to attend scheduled clinic appointments, adhere to dose 
   titration schedule as directed
- agree to use no other cannabis based products for the duration of the trial
- understand it is illegal to drive whilst taking THC containing cannabis products, to take 
  cannabinoid products outside of Australia or to endorse legal documents whilst taking THC 
  containing cannabis products
- provide fully informed consent
*physician assessed

Minimum age25 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

Patients with:

- a history of hypersensitivity to any cannabinoid product
- unstable untreated cardiovascular disease (hypertension, ischemic heart disease, congestive 
  cardiac failure)
- severe hepatic impairment (total bilirubin >1.5 times the upper limit of the institution's normal 
  range. Asparate aminotransferase (AST), and alanne aminotransferase (ALT) >3.0 time the upper 
  limit of the institution's normal range; subjects with liver metastasis may have an AST and ALT of 
  >5.0 times the upper limit of normal
- severe renal impairment (eGFR <20mls/min/1.73m2)
- history of psychiatric disorders (severe depression or anxiety, personality disorder, psychosis, 
  schizophrenia, first degree relative with schizophrenia and/or suicidal ideation)
- cognitive impairment (SLUMS - St Louis University Mental Status) examination <20/30
- known substance use disorder (ASSIST - Alcohol, Smoking and Substance Involvement Screening 
  Test) examination score >27+ 
- history that drug diversion may be a risk for them or their family/carers
- females who are pregnant or lactating
- concurrent or participation of a new clinical entity with the last 28 days
- treatment with a new specific anticancer agent (chemotherapy, targeted or hormonal therapy) or 
  radiation within the last 7 days
Contact details and further information

Sponsor Primary Sponsor Type: Hospital
Primary Sponsor Name: Mater Misericordiae Limited
Primary Sponsor Address: Mater Misericordiae Ltd Raymond Terrace South Brisbane Qld 4101
Primary Sponsor Country: Australia

Trial IDACTRN12619000037101

Contact person for information and recruitmentMrs
Georgie Huggett
Clinical Trial Coordinator Palliative and Supportive Care Mater Misericordiae Ltd Raymond Terrace South Brisbane, Qld 4101
+61 7 3163 6057
+61 7 6163 1588
Further information icongeorgie.huggett@mater.org.au
Australia

Clinical study Of caNNabidiol in children and adolesCenTs with Fragile X Open-Label Extension (CONNECT-FX OLE)

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Trial Information

Trial summary

This study is evaluating the long term and safety and tolerability of ZYN002, a clear gel that can be applied to the skin (called transdermal application) twice a day for treatment of symptoms of Fragile X Syndrome (FXS)

Who is it for?
Patients who have been diagnosed with Fragile X Syndrome and are aged between 3 and 18 years old who have participated in the ZYN2-CL-016 double-blind study and meet certain eligibility criteria.

Study details
Eligible participants will undergo up to a 52-week treatment period. Participants who are taking anti-epileptic drugs may undergo an additional 1-2 weeks of treatment to taper off study drug treatment.  All participants will be assigned to ZYN002. Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders. 

Participants whose weight changes during the course of the study may have their doses changed at the investigator’s discretion on or after the Month 1 visit, or reduced due to tolerability issues at investigator’s discretion.

Blood samples will be collected for safety analysis of ZYN002.   Additionally, the parents/caregivers will be asked to complete some questionnaires. There will be other questionnaires and scales that will be completed at the site by the study doctor and/or with the participant and their parents/caregivers.

Participation in this study may help the child’s/ adolescent’s FXS symptoms; however, we cannot guarantee that he/ she will get any benefits from this study. The results of this study may benefit future patients.

Broad Health ConditionFragile X Syndrome

Specific Health ConditionHuman Genetics and Inherited Disorders
Other human genetics and inherited disorders
Neurological
Other neurological disorders

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,QLD,VIC

Hospital
Lady Cilento Children's Hospital - South Brisbane

Hospital
Fragile X Alliance Inc. - Caulfield North

Hospital
The Children's Hospital at Westmead - Westmead

Postcode
4101 - South Brisbane

Postcode
3161 - Caulfield North

Postcode
2145 - Westmead

Trial location outside Australia

Country
United States of America

State
Multiple Sites

Country
New Zealand

State
Wellington

Anticipated date of last participant enrolment30/09/2019

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Participated in a certain number of visits in the ZYN2-CL-016 study
2.	Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
3.	Patients and parents/caregivers must be adequately informed of the nature, risks of the study, and give written informed consent prior to enrollment in ZYN2-CL-017.
4.	In the Investigator’s opinion, the patients and parents/caregivers are reliable and are willing and able to comply with all protocol requirements and procedures.
5.	Females of childbearing potential must have a negative pregnancy test at all designated visits

Minimum age3 Years

Maximum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1.	Patient is receiving any investigational drugs (not ZYN002) or using any experimental devices.
2.	Patient has an ongoing serious adverse event (SAE) or has experienced a SAE in ZYN2-CL-016, which in the opinion of the Investigator, should exclude them from participation. 
3.	Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of trial drug. 
4.	Patients who have alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels = 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels = 3 times the ULN as determined from patient safety laboratories.
Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals Pty. Ltd
Primary Sponsor Address: 2 Riverside Quay Soutbank, VIC 3006
Primary Sponsor Country: Australia

Trial IDACTRN12618001868279

Contact person for information and recruitmentDr
Nancy R. Tich
Zynerba Pharmaceuticals Inc.,80 West Lancaster Ave., Suite 300, Devon, PA 19333
+1-973-727-4117

Further information iconTichn@zynerba.com
United States of America

A Randomised, Double Blind, Placebo-Controlled Trial of Medicinal Cannabis in Adults with Tourette's Syndrome

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Trial Information

Trial summary

Tourette’s syndrome (TS) is a neurological disorder that develops in childhood and often continues into adulthood. Individuals with TS make involuntary movements and vocalisations (known as tics), which may be painful, embarrassing and functionally impairing. There is no cure for TS, but certain medications (particularly antipsychotics) are effective at suppressing tics when taken continuously. Unfortunately, these medications have significant and independently-disabling side effects, which limit their acceptability for many sufferers. 

The human body has its own (‘endogenous’) cannabinoid neurotransmitter system, which facilitates communication between nerve cells in the brain. This system is implicated in the control of normal movement and the development of movement disorders such as TS. The ‘endocannabinoid’ system could therefore be a new therapeutic target for tic suppression. There is some early evidence to support the effectiveness of cannabinoids in TS, but well-designed clinical trials have yet to be conducted. 

We plan to use an oral formulation of medicinal cannabis containing two cannabinoids: tetrahydrocannabinol and cannabidiol. This formulation does not intoxicate or cause the unpleasant psychiatric effects of ‘street’ cannabis. 

We predict that treatment with tetrahydrocannabinol and cannabidiol will significantly reduce tics, when compared to placebo, as well as improving psychiatric and cognitive symptoms associated with TS. We also predict that this formulation will be well tolerated, without psychiatric side effects, and will not lead to craving or intoxication. Therefore, this study may support the development of a new, safe and effective treatment for TS, with potential applications to other neurological disorders. 

Broad Health ConditionTourette's Syndrome

Specific Health ConditionNeurological
Other neurological disorders

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Anticipated date of first participant enrolment21/01/2019

Anticipated date of last participant enrolment31/07/2021

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

Confirmed diagnosis of Tourette's syndrome with a tic severity score greater than 20 on the Yale Global Tic Severity Scale. 
Must agree not to drive a motor vehicle during the trial.
Able to give written and informed consent.

Minimum age18 Years

Maximum age70 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

History of epilepsy.
History of multiple sclerosis.
History of an additional movement disorder such as Parkinson's disease.
History of dementia..
Pregnancy and breastfeeding.
Recreational cannabis smoker (participants must have a negative drug screen at study entry).
Current treatment with deep brain stimulation.
Current drug or alcohol abuse (excluding tobacco).
Any history of psychosis.
Diagnosis of bipolar disorder.
History of any previous high-lethality suicide attempt, history of any suicide attempt in the last 12-months or at high-risk of suicide in the opinion of the screening clinician. 
Currently using antipsychotic or dopamine-depleting agents as treatment for Tourette's syndrome. Participants who have undergone a successful 4 week washout of current exclusionary therapies overseen by the Investigator will be eligible.
Allergy to tetrahydrocannabinol and cannabidiol or the tablet excipients.
Taken another study (experimental) drug within the past 30 days.
Contact details and further information

Sponsor Primary Sponsor Type: Hospital
Primary Sponsor Name: Wesley Medical Research Insititute
Primary Sponsor Address: Level 8, East Wing The Wesley Hospital 451 Coronation Drive Auchenflower QLD 4066
Primary Sponsor Country: Australia

Trial IDACTRN12618000545268

UTNU1111-1211-3260

Contact person for information and recruitmentDr
Philip Mosley
Neurosciences Queensland Level 1, St Andrews Place 33 North Street Spring Hill QLD 4000
+61738393688
+61738393588
Further information iconp.mosley@uq.edu.au
Australia

Clinical study of Cannabidiol in children and adolescents with Developmental and Epileptic Encephalopathy

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Trial Information

Trial summary

A sequential, multi-stage, open-label, multi-national, multiple-center, multiple-dose study to assess the long-term safety and tolerability of ZYN002 (transdermal CBD gel) in child and adolescent epilepsy patients 3 to <18 years of age with seizures associated with developmental and epileptic encephalopathies (DEE) according to the International League Against Epilepsy (ILEA) classification.
In Period A patients will undergo a baseline period of 4-weeks, followed by a 2-week titration period, and a 24-week maintenance period. Patients will be treated for a total of 26 weeks. For Patients not continuing to Period B, following Week 26 or Early Termination, study drug will be tapered over a 1 to 3-week period (depending on dose). After the final dose, patients will be followed weekly for 4 weeks by telephone.  After the 4 weeks, the patient will be discharged from the study. 
Patients progressing to Period B will continue to receive ZYN002 for a further 24 weeks at the same maintenance dose received at Week 26 (e.g. end of Period A). Upon treatment termination, the patient will be required to complete the taper and follow-up period.  After the final tapered dose, patients will be followed weekly for 4 weeks by telephone.  After the 4 weeks of follow-up, the patient will be discharged from the study.

Broad Health ConditionEpilepsy
Developmental disability

Specific Health ConditionNeurological
Epilepsy
Neurological
Other neurological disorders

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
VIC

Hospital
Austin Health - Austin Hospital - Heidelberg

Postcode
3084 - Heidelberg

Trial location outside Australia

Country
New Zealand

State
Wellington

Anticipated date of first participant enrolment9/04/2018

Anticipated date of last participant enrolment14/12/2018

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1. Male or female, 3 to  less than 18 years of age, inclusive, at the time of screening.
2. Judged by the Investigator to be in generally good health at the Screening Visit based upon the results of a medical history, physical examination, 12-lead ECG, and clinical laboratory test results. 
3. Patients must have a diagnosis of developmental and epileptic encephalopathy (DEE) as defined by the International League Against Epilepsy Classification (Scheffer 2017) with generalized motor, focal motor and focal impaired awareness or focal bilateral to tonic-clonic seizures. Examples of DEE that may be included, but not limited to: Lennox-Gastaut Syndrome, Dravet Syndrome, West Syndrome/ Infantile Spasms and Doose Syndrome. The diagnosis must be established for 1 years and documented by review of electroencephalogram (EEG), , and or genetic testing if available, and narrative from the physician who manages the patient’s epilepsy. The patient/legally authorized representation may elect to have the patient participate in genetic seizure panel testing to characterize a genetic diagnosis.
4. Patient must experience a threshold number of generalized motor seizures (i.e. tonic, clonic, atonic, tonic-clonic seizures, and generalized tonic-clonic), focal motor, focal impaired awareness or focal to bilateral tonic-clonic seizures during the Baseline period.
5. Patient is currently being treated and maintained with a stable regimen of between one (1) and four (4) AEDs for at least 4 weeks before screening, and willing to maintain a stable regimen during the treatment period. If a benzodiazepine is used as a rescue medication greater than 2 times per week, it will be counted as an AED. Patient taking ethosuximide, felbamate and vigabatrin must be on stable therapy for at least 6 months. Patient taking felbamate must have had no clinically relevant changes in hematology or liver function.
6. Patient has history of developmental delay with regression, slowing or plateau in at least one developmental domain. 
7. All interventions for epilepsy must be stable for at least one month prior to screening. 
8. Patient/caregiver is able and willing to maintain daily diaries for seizures, daily skin assessments and good day/bad day assessment.
9. Patient has a body mass index between 13 and 35 kg/m2 and weighs no less than 12 kg.
10. Sexually active females of childbearing potential must use an acceptable method of contraception. Acceptable methods of contraception include: hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, vasectomy, and intrauterine device.
11. Females of childbearing potential must have a negative pregnancy test at the Screening Visit, as well as at Day 1. 
12. Patient is reasonably stable medically and is unlikely to require changes in drug therapy during the Treatment Period of the study, or interfere with the objectives of the study, or the ability to adhere to protocol requirements.
13. Patient/caregiver/legally authorized representative must be adequately informed of the nature and risks of the study and give written informed consent prior to screening.
14. In the Investigator’s opinion, the patient and/or caregiver is reliable and is willing and able to comply with all protocol requirements and procedures.

Minimum age3 Years

Maximum age17 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1. Patient has a history of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any adhesives, compound, or chemical class related to ZYN002 or its excipients.
2. Patient has been exposed to any investigational drug or device less than 30 days prior to screening or plans to take another investigational drug at any time during the study.
3. Patient has used cannabis or any CBD- or THC-containing product within 12 weeks of the Screening Visit or during the study.
4. Patient on the following AEDs for less than 6 months: ethosuximide, felbamate and vigabatrin.
5. Patient has had a change in AED regimen in the 4 weeks prior to screening. 
6. Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels equal to 3x the upper limit of normal (ULN) as determined from Screening safety laboratories.
7. Patient/caregiver demonstrates behavior indicating unreliability or inability to comply with the requirements of the protocol. 
8. Patient has had a change in epilepsy dietary therapy in the 4 weeks prior to screening.
9. Patient has seizures secondary to illicit drug or alcohol use. 
10. Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including the following medications: midazolam, oral ketoconazole, fluconazole, nefazodone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxel, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinoin, vincristine, vinorelbine and St. John’s Wort.
11. Patient has a history of suicide attempt in the last 5 years or more than one lifetime suicide attempt. 
12. Patient responds “yes” to Question 4 or 5 of C-SSRS (Children) during Screening and at any time during the study.
13. Patient has a positive result for the presence of Hepatitis B surface antigen (HBsAg), Hepatitis C virus antibodies (HCV-Ab), or human immunodeficiency virus (HIV) antibodies.
14. Patient has any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study treatment.
15. Patient has known history of cardiac disease.  
16. Patient has any skin disease or condition, including eczema (on shoulders/arms, back or thighs), psoriasis, melanoma, acne (on shoulders/arms/back or thighs), contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may affect treatment application, application site assessments, or absorption of the study drug.

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals Pty Ltd
Primary Sponsor Address: Zynerba Pharmaceuticals Pty Ltd Offices of Pricewaterhouse Coopers 2 Riverside Quay Southbank VIC 3006
Primary Sponsor Country: Australia

Trial IDACTRN12618000516280

Contact person for information and recruitmentDr
Nancy R. Tich, Ph.D.
Zynerba Pharmaceuticals Pty, Ltd. 80 West Lancaster Ave., Suite 300, Devon, Pennsylvania
+1-973-727-4117

Further information icontichn@zynerba.com
United States of America

CannabisCINV: A placebo-controlled trial evaluating an oral THC/CBD cannabis extract for secondary prevention of chemotherapy-induced nausea and vomiting in patients of any known malignancy receiving chemotherapy.

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Trial Information

Trial summary

The primary purpose of this trial is to evaluate the efficacy of an oral capsule containing plant-derived tetrahydrocannbinol (THC) and cannabidiol (CBD) for the prevention of chemotherapy-induced nausea and vomiting (CINV). 

Who is it for?
You may be eligible to participate in this trial if you are aged 18 or over and have been diagnosed with any cancer for which you are scheduled to receive at least three further chemotherapy cycles using intravenous chemotherapy of high or moderate emetic risk.

Study details
We will first enrol 80 participants in a pilot trial and if the data from these participants shows the medicine works and is well tolerated, a further 170 participants will be enrolled, this is called the definitive trial.

Participants enrolled in the pilot trial will be randomly allocated (by chance) to receive the study drug for the first five days of their first chemotherapy cycle following enrolment, followed by a placebo capsule for the first five days of the next chemotherapy cycle, or to receive the placebo first followed by the study treatment. All participants will then choose which treatment they would prefer to take for the first five days of the third chemotherapy cycle. Participants will be asked to complete a number of questionnaires relating to their nausea and vomiting, quality of life and any side effects of the treatment. 

Participants enrolled in the definitive trial will be randomly allocated (by chance) to receive the study drug or the placebo for the first five days of their next three chemotherapy cycles following enrolment, Participants will be asked to complete a number of questionnaires relating to their nausea and vomiting, quality of life and any side effects of the treatment. 

It is hoped that this trial will provide preliminary information on the efficacy of THC and CBD capsules for the prevention of CINV, which will inform further clinical trials.

Broad Health ConditionChemotherapy induced nausea and vomiting
Cancer

Specific Health ConditionCancer
Any cancer

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,QLD,SA

Hospital
The Chris O’Brien Lifehouse - Camperdown

Hospital
Concord Repatriation Hospital - Concord

Hospital
Coffs Harbour Base Hospital - Coffs Harbour

Hospital
Wollongong Hospital - Wollongong

Hospital
Orange Health Service - Orange

Hospital
St George Hospital - Kogarah

Hospital
Royal North Shore Hospital - St Leonards

Hospital
Campbelltown Hospital - Campbelltown

Hospital
The Tweed Hospital - Tweed Heads

Hospital
Riverina Cancer Care Centre - Wagga Wagga

Hospital
Southern Highlands Private Hospital - Bowral

Hospital
Gosford Hospital - Gosford

Hospital
Flinders Medical Centre - Bedford Park

Hospital
Gold Coast University Hospital - Southport

Hospital
Sydney Adventist Hospital - Wahroonga

Hospital
John Flynn - Gold Coast Private Hospital - Tugun

Postcode
2050 - Camperdown

Postcode
2139 - Concord Repatriation Hospital

Postcode
2450 - Coffs Harbour

Postcode
2500 - Wollongong

Postcode
2800 - Orange

Postcode
2217 - Kogarah

Postcode
2065 - St Leonards

Postcode
2560 - Campbelltown

Postcode
2485 - Tweed Heads

Postcode
2650 - Wagga Wagga

Postcode
2576 - Bowral

Postcode
2250 - Gosford

Postcode
5042 - Bedford Park

Postcode
4215 - Southport

Postcode
2076 - Wahroonga

Postcode
4224 - Tugun

Anticipated date of first participant enrolment30/11/2016

Anticipated date of last participant enrolment30/10/2021

Phase of TrialPhase 2 / Phase 3

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1.	Adults, aged 18 years and older, with known malignancy of any stage
2.	Receiving intravenous chemotherapy of high or moderate emetic risk as defined by MASCC criteria on Treatment Day 1 administered in cycles of planned duration greater than or equal to 14 days and less than or equal to 21 days
Note:
-	Chemotherapy agents classified as low and/or minimal emetic risk may be used concurrently throughout the treatment period (acceptable combination regimens include but not limited to: FOLFOX, carboplatin day 1 and gemcitabine day 1 and 8)
-	Multi-day use of chemotherapy of high or moderate emetic risk is permitted up until but not beyond day 5 is permitted, when the continuation of the chemotherapy is part of the overall Day 1 regimen (acceptable multi-day regimens include but not limited to: BEP, TIP; unacceptable multi-day regimens include but not limited to: weekly cisplatin, weekly carboplatin, cisplatin day 1 & 8 with gemcitabine day 1 & 8, MVAC)
-	Concurrent oral chemotherapy and radiotherapy are not permitted 
3.	Requires greater than or equal to 2 further cycles of chemotherapy
4.	Experiencing significant CINV during previous cycle 1
-	defined as need for rescue medications for vomiting or distress by nausea, and/or greater than or equal to moderate nausea on 5-point rating scale, despite best-practice MASCC guideline-consistent anti-emetic regimen
5.	ECOG performance status of 0, 1 or 2
6.	Predicted life expectancy of greater than or equal to 4 months
7.	Willing and able to comply with all study requirements, including treatment, timing and nature of required assessments including diary, quality of life forms, urine tests, and any mandated blood tests
8.	Signed, written informed consent

Minimum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1.	Symptomatic primary or secondary CNS malignancy
2.	Symptomatic gastrointestinal obstruction
3.	Disease-related nausea or vomiting requiring daily anti-emetic therapy
4.	Unstable cardiovascular disease (uncontrolled hypertension, unstable ischaemic heart disease, unstable congestive cardiac failure)
5.	History of epilepsy or recurrent seizures
6.	History of schizophrenia, other psychotic illness, severe personality disorder, suicidal ideation, or other significant psychiatric disorder, other than depression associated with underlying condition
7.	Substance use disorder (ICD-10 criteria (abuse, dependence) to alcohol, opioids, benzodiazepines, or illicit stimulants
8.	Serious medical or psychiatric condition that might limit the ability of the patient to consent to the study and/or comply with the protocol
9.	Scheduled to receive oral chemotherapy during planned duration of study
10.	Patient has received or is scheduled to receive radiation therapy to the brain, abdomen or pelvis in the week prior to commencement of study treatment, or during study treatment
11.	Is scheduled to receive any investigational drug during the present study
12.	Patients using or having used cannabis or cannabinoid based medications within 30 days of study entry and unwilling to abstain for the duration of the study
13.	Prior hypersensitivity or intolerable adverse reaction to cannabis or cannabinoid based medications, 5HT3 antagonist, dexamethasone, NK1 antagonist
14.	Unwilling to avoid driving or operating machinery during and for 72 hours after taking study medication
15.	Concerns regarding safe storage of study medication (eg. unsuitable home environment)
16.	Pregnancy, lactation, or inadequate contraception. Women must be post-menopausal, infertile, or use a reliable means of contraception. Women of childbearing potential must have a negative pregnancy test done within 7 days prior to registration. Men must have been surgically sterilised or use a (double if required) barrier method of contraception.
17. Patients who were previously enrolled in this study and received the study intervention (oral THC/CBD and/or placebo)
18. Patients who declare they have been convicted of a criminal offence.
Contact details and further information

Sponsor Primary Sponsor Type: University
Primary Sponsor Name: University of Sydney
Primary Sponsor Address: Campderdown NSW 2050
Primary Sponsor Country: Australia

Trial IDACTRN12616001036404

Contact person for information and recruitmentMs
Trial Coordinator
Organisation: University of Sydney NHMRC CTC, Level 6 Chris O Brien Lifehouse, 119–143 Missenden Road, Camperdown NSW 2050
+61295625000

Further information iconcannabisCINV@ctc.usyd.edu.au
Australia

Medicinal Cannabis for Anorexia in Advanced Cancer

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Trial Information

Trial summary

The primary purpose of this trial is to evaluate whether a vaporised form of medicinal cannabis is feasible and effective in increasing appetite in cancer patients with anorexia. 
Who is it for? You may be eligible for this trial if you are aged 18 or over, have an advanced cancer and have been suffering from anorexia for at least two weeks. 
Study details: Participants will receive a range of doses of vaporised botanical cannabis flower bud one hour before your meals (three times a day) for 7 days unless side effects occur. Each dose may be different from the previous dose, but they will all be made up to the same weight of plant material by adding different amounts of inactive plant material (‘placebo’, which has the same characteristics of leaf cannabis with the active components removed).   Participants will have blood samples taken at multiple timepoints up to 4 hours following each morning dose and will complete a number of questionnaires and a daily food record to determine the concentration of the drug in the blood, and to monitor its effect on appetite, mood and other factors. 
It is hoped that the findings from this trial will provide information on whether inhalation of medicinal cannabis is feasible for achieving a safe and effective blood concentration for the treatment of anorexia in cancer patients.

Broad Health ConditionCancer
Anorexia

Specific Health ConditionCancer
Any cancer

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW

Hospital
Sacred Heart Hospice - Darlinghurst

Hospital
St Vincent's Hospital (Darlinghurst) - Darlinghurst

Postcode
2010 - Darlinghurst

Anticipated date of first participant enrolment25/07/2016

Anticipated date of last participant enrolment31/12/2019

Phase of TrialPhase 1 / Phase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

1) Age 18 years or above;
2) Advanced cancer; 
3) Anorexia for at least 2 weeks (defined as numeric rating scale [0 no appetite – 10 best possible appetite] score less than or equal to 4) unresponsive to the optimisation of treatment of causative medical conditions
4) English-speaking (or have an interpreter available);
5) Performance status (Australia-modified Karnofsky Scale score) of 40 or above;
6) Written informed consent.

Minimum age18 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

1) Hepatic impairment (Child’s Stage B)
2) Renal impairment (estimated glomerular filtration rate of <10 mL/min)
3) Cognitive impairment (Montreal Cognitive Assessment (MOCA score<26); 
4) Psychiatric disorders (severe depression or anxiety, personality disorder, history of psychosis, schizophrenia, and/or suicidal ideation); 
5) Acute delirium or delirium within < 30 days; 
6) Unstable cardiovascular disease (uncontrolled hypertension, unstable ischaemic heart disease, unstable congestive cardiac failure); 
7) Impaired pulmonary function which prohibits use of vaporiser; 
8) Prior adverse reaction to botanical cannabis/pharmaceuticals containing cannabinoids; 
9) Pregnant, breastfeeding or unwillingness to use oral contraceptives; 
10) Substance use disorder (ICD-10 criteria (abuse, dependence)) to alcohol, opioids, benzodiazepines or simulants (excluding caffeine, tobacco). 
11) Recent use of cannabis or cannabinoids within < 30 days (based on self-report and urine drug screen at eligibility).
12) Prescribed opioid, benzodiazepine, antidepressant, antipsychotic, corticosteroid, progestin, omega fatty acids and/or dietary supplements, which do not meet the criteria for therapies allowed at eligibility assessment 
13) Participation in a clinical trial of another chemical entity.
14) Conditions causing irreversible or blood transfusion dependent anaemia where the volume of blood sampling required for this study is contraindicated in the opinion of the treating clinician.
Contact details and further information

Sponsor Primary Sponsor Type: University
Primary Sponsor Name: University of New South Wales
Primary Sponsor Address: Anzac Parade, Kensington NSW 2052
Primary Sponsor Country: Australia

Trial IDACTRN12616000516482

Contact person for information and recruitmentProf
Meera Agar
Ingham Institute of Applied Medical Research 1 Campbell Street, Liverpool NSW 2170
+61 0295144232
+61-2-8738 9149
Further information iconmeera.agar@sswahs.nsw.gov.au
Australia

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome

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Trial Information

Trial summary

To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel
formulation, for up to 38 weeks, in patients ages 4 to < 18 years, in the treatment of
22q.11.2 Deletion Syndrome (22qDS).

Broad Health Condition22qDS
Chromosome 22q11.2 deletion syndrome
Autosomal dominant Opitz G/BBB syndrome
CATCH22
Cayler cardiofacial syndrome
Conotruncal anomaly face syndrome (CTAF)
Shprintzen syndrome
Velo-cardio-facial syndrome (VCFS)
Velocardiofacial syndrome

Specific Health Condition22Q Deletion Syndrome

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
QLD,VIC,

Hospital
Lady Cilento Children's Hospital - South Brisbane

Phase of TrialPhase 1/Phase 2

Eligibility

Key inclusion criteria

Inclusion Criteria:

    1. Male or female children and adolescents aged 4 to less than 18 years, at the time of
       Screening.

    2. Judged by the Investigator to be in generally good health at Screening based upon the
       results of a medical history, physical examination, and clinical laboratory test
       results.

    3. Patients must have a diagnosis of 22q Deletion Syndrome (22qDS) confirmed by genetic
       testing.

    4. Patients have a Clinical Global Impression-Severity (CGI-S) score of 4 or higher at
       Screening and Visit 2.

    5. Patients must have a Pediatric Anxiety Rating Scale-Revised (PARS-R) severity score of
       10 or higher at Screening and Visit 2.

    6. Patients with a history of seizure disorders must currently be receiving treatment
       with a stable regimen of one or two AEDs, or must be seizure-free for one year if not
       currently receiving AEDs.

    7. If patients are receiving non-pharmacological behavioral and/or dietary interventions,
       they must be stable for three months prior to Screening.

    8. Patient has demonstrated stable calcium levels for one year prior to Screening.

    9. Patients have a body mass index between 12-35 kg / m2 (inclusive).

   10. Females of childbearing potential must have a negative pregnancy test at the Screening
       Visit and a negative pregnancy test at all designated study visits.

   11. Patients and parents/caregivers agree to abide by all study restrictions and comply
       with all study procedures.

   12. Patients and parents/caregivers must be adequately informed of the nature and risks of
       the study and give written informed consent (and assent if applicable) prior to
       Screening.

   13. Parents/caregiver(s) must provide written consent to assist in study drug
       administration.

   14. In the Investigator's opinion, patients and parents/caregivers are reliable and
       willing and able to comply with all protocol requirements and procedures.

  Exclusion Criteria:

    1. Females who are pregnant, nursing, or planning a pregnancy; females of childbearing
       potential and male patients with a partner of childbearing potential who are unwilling
       or unable to use an acceptable method of contraception for the duration of therapy and
       for three months after the last dose of study medication.

    2. History of significant allergic condition, significant drug-related hypersensitivity,
       or allergic reaction to any compound or chemical class related to ZYN002 or its
       excipients.

    3. Exposure to any investigational drug or device = 30 days prior to Screening or at any
       time during the study.

    4. Patient has ALT, AST, or total bilirubin levels = 2 times the ULN)or has alkaline
       phosphatase levels = 3 times the ULN as determined from Screening safety laboratories.

    5. Use of cannabis or any THC or CBD-containing product within three months of Screening
       Visit or during the study.

    6. Patient has a positive drug screen.

    7. Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate,
       carbamazepine, phenytoin or vigabatrin.

    8. Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for
       CYP3A4 including but not limited to the following medications: midazolam, oral
       ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone,
       cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl,
       halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide,
       quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin,
       vincristine, vinorelbine, St. John's Wort, and grapefruit juice/products.

    9. Patient with diagnosis of known genetic disorder, other than 22qDS.

   10. Patient has diagnosis of DiGeorge or Velocardiofacial syndrome without the presence of
       22qDS.

   11. Patient has a primary psychiatric diagnosis other than 22qDS or anxiety, including
       bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder.

   12. Patient is on stable treatment of >6 months of not more than two psychoactive
       medications at screening or throughout the study (with the exception of one
       psychoactive medication prescribed for sleep).

   13. Patient has an advanced, severe, or unstable disease that may interfere with the study
       outcome evaluations.

   14. Patient is expected to initiate or change pharmacologic or non-pharmacologic
       interventions during the course of the study.

   15. Patient has an acute or progressive neurological disease, or any psychiatric disorder
       or severe mental abnormalities that are likely to require changes in drug therapy or
       interfere with the objectives of the study or ability to adhere to protocol
       requirements.

   16. Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.

   17. Patients at risk of needing cardiovascular surgical repair within the upcoming 12
       months.

   18. Patient has unstable cardiovascular disease, such as advanced arteriosclerosis,
       cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac
       conduction problems, exercise-related cardiac events including syncope and
       pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure,
       hypokalemia, family history of Long QT Syndrome), other serious or other clinically
       unstable cardiac problems as indicated by history, physical examination, or ECG.

   19. Any clinically significant condition or abnormal findings at the Screening Visit that
       would, in the opinion of the Investigator, preclude study participation or interfere
       with the evaluation of the study medication.

   20. Any skin disease or condition that may affect treatment application, application site
       assessments, or absorption of the study drug.

   21. History of treatment for, or evidence of, drug abuse within the past year.

   22. Patient responds "yes" to Question '4' or '5' on the Columbia Suicide Severity Rating
       Scale Children (C-SSRS) during Screening or at any time on study.

Minimum age4 Years

Maximum age17 Years

GenderAll

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals, Inc.

Trial websitehttps://clinicaltrials.gov/show/NCT05149898

Trial IDNCT05149898








Clinical Study of Cannabidiol in Children and Adolescents With Fragile X Syndrome

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Trial Information

Trial summary

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the
efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children and
adolescent patients with Fragile X Syndrome (FXS). Eligible participants will participate in
up to an 18 week treatment period, where all participants will receive placebo or active
study drug. Patients ages 3 to < 18 years, will be eligible to participate.

Broad Health ConditionHuman Genetics and Inherited Disorders
Other human genetics and inherited disorders

Specific Health ConditionFragile X Syndrome

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,QLD,VIC,

Hospital
Westmead Children's Hospital

Hospital
Lady Cilento Children's Hospital - South Brisbane

Phase of TrialPhase 3

Eligibility

Key inclusion criteria

Inclusion Criteria:

    -  Male or female children and adolescents aged 3 to < 18 years, at the time of
       Screening.

    -  Judged by the Investigator to be in generally good health at Screening based upon the
       results of medical history, physical exam, 12-lead ECG and clinical laboratory test
       results. -Laboratory results outside the reference range must be documented as not
       clinically significant by both the Investigator and Sponsor.

    -  Participants must have a diagnosis of FXS through molecular documentation of full
       mutation of the FMR1 gene documented through genetic testing at Screening.

    -  Patients with a history of seizure disorders must currently be receiving treatment
       with a stable regimen of no more than two anti-seizure medications (ASMs) for the four
       weeks preceding study Screening; or must be seizure-free for one year if not currently
       receiving ASMs.

    -  Patients taking psychoactive medication(s) should be on a stable regimen of not more
       than three such medications for at least fours weeks preceding Screening and must
       maintain that regimen throughout the study. Psychoactive medications include (but are
       not limited to) antipsychotics, antidepressants, anxiolytics, attention-deficit /
       hyperactivity disorder (ADHD) medications, and medications for sleep.

    -  If patients are receiving non-pharmacological, behavioral and/or dietary
       interventions, they must be stable and have been doing so for three months prior to
       screening.

    -  Patients have a body mass index between 12-30 kg/m2 (inclusive).

    -  Females of childbearing potential must have a negative serum pregnancy test at the
       Screening Visit and a negative serum or urine pregnancy test at all designated visits.

    -  Patients and parents/caregivers must be adequately informed of the nature and risks of
       the study and given written informed consent prior to Screening.

    -  Patients and parents/caregivers agree to abide by all study restrictions and comply
       with all study procedures, and in the Investigator's opinion, are reliable and willing
       and able to comply with all protocol requirements and procedures.

  Exclusion Criteria:

    -  Females who are pregnant, nursing or planning a pregnancy; females of childbearing
       potential and male patients with a partner of childbearing potential who are unwilling
       or unable to use an acceptable method of contraception as outlined below for the
       duration of therapy and for three months after the last dose of study medication.
       Standard acceptable methods of contraception include abstinence (defined as refraining
       from heterosexual intercourse from screening to three months after the last dose of
       study medication) or the use of a highly effective method of contraception, including
       hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, spermicide,
       vasectomy, or intrauterine device. The reliability of sexual abstinence needs to be
       evaluated in relation to the duration of the clinical trial and the preferred and
       usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal,
       post-ovulation methods) is not an acceptable method of contraception.

    -  History of significant allergic condition, significant drug-related hypersensitivity,
       or allergic reaction to any compound or chemical class related to ZYN002 or its
       excipients.

    -  Exposure to any investigational drug or device less than or equal to 30 days prior to
       Screening or at any time during the study.

    -  Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin
       levels greater than or equal to 2 times the upper limit of normal or alkaline
       phosphatase levels greater than or equal to 3 times the upper limit of normal.

    -  Use of cannabis or any THC or CBD-containing product within 3 months of Screening
       Visit or during the study (aside from ZYN002).

    -  Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates,
       amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable
       administered for blood draws and ECG collection), and opiates.

    -  Patient is using the following AEDs (medications for the treatment of seizures and/ or
       epilepsy): clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin,
       or vigabatrin.

    -  Patient is using a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4
       including but not limited to the following medications: midazolam (except single doses
       administered for the purposes of obtaining blood samples and ECG's), oral
       ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone,
       cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl,
       halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide,
       quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin,
       vincristine, vinorelbine, St. John's Wort, and grapefruit Juice/products.

    -  Patients may not be taking any benzodiazepines (except single doses administered for
       the purposes of obtaining blood samples and ECGs) at screening or throughout the
       study.

    -  Patient is expected to initiate or change pharmacologic or non-pharmacologic
       interventions during the course of the study.

    -  Patient has an advanced, severe, or unstable disease that may interfere with the study
       outcome evaluations.

    -  Patient has acute or progressive neurological disease, psychosis, schizophrenia or any
       other psychiatric disorder or severe mental abnormalities (other than FXS) that are
       likely to require changes in drug therapy or interfere with the study objectives or
       ability to adhere to protocol requirements.

    -  Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.

    -  Patient has known history of cardiovascular disease, advanced arteriosclerosis,
       structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities,
       coronary artery disease, cardiac conduction problems, exercise-related cardiac events
       including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g.,
       heart failure, hypokalemia, family history of Long QT Syndrome), or other serious
       cardiac problems.

    -  Any clinically significant condition or abnormal findings at the Screening Visit that
       would, in the opinion of the Investigator, preclude study participation or interfere
       with the evaluation of the study medication.

    -  Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact
       dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may
       affect treatment application, application site assessments or absorption of the trial
       drug.

    -  History of treatment for, or evidence of, drug abuse within the past year.

    -  Previous participation in a ZYN002 study (with the exception of patients who were
       screen failures in Study ZYN2-CL-016 and did not enter Study ZYN2-CL-017).

    -  Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or
       at any time on study.

Minimum age3 Years

Maximum age17 Years

GenderAll

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals, Inc.

Trial websitehttps://clinicaltrials.gov/show/NCT04977986

Trial IDNCT04977986








Evaluation of the Effectiveness of Cannabidiol in Treating Severe Behavioural Problems in Children and Adolescents With Intellectual Disability

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Trial Information

Trial summary

This is a multi-site, double-blind, parallel group, randomized, placebo-controlled study of
140 participants comparing oral purified cannabidiol isolate (CBD) with placebo in reducing
Severe Behavioral Problems (SBP) at 8 week in children aged 6 - 18 years with Intellectual
Disability (ID). Eligible participants will be randomized 1:1 to receive either CBD or
placebo.

Broad Health Conditionpediatric
intellectual disability
severe behavior problems

Specific Health ConditionIntellectual Disability
Child Behavior Problem

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,VIC,

Hospital
The Children's Hospital at Westmead

Hospital
Monash Children's Hospital

Hospital
Royal Children's Hospital / Murdoch Children's Research Institute

Phase of TrialPhase 2

Eligibility

Key inclusion criteria

Inclusion Criteria:

    1. Males and females aged 6 - 18 years of age;

    2. DSM-5 diagnosis of intellectual disability (ID):

         1. Full scale IQ < 70 on standardized cognitive assessment. Testing results must be
            sighted by the investigators and performed within two years of enrollment. In the
            event that records of prior testing are unavailable or the assessment was more
            than 2 years prior, IQ will be estimated using the Wechsler Abbreviated Scale of
            Intelligence-II.

         2. Deficit in adaptive function (basis for severity rating of ID in DSM-5) in at
            least one activity of life on the Vineland Adaptive Behavior Scales (derives
            scores in Communication, Daily Living Skills and Socialization domains, and a
            Global Adaptive score). If records of prior testing are unavailable or the
            assessment was more than 2 years prior, this will be completed by the parent or
            guardian.

    3. SBP: Defined as scores of:

         1. 18 or higher on the Aberrant Behavior Checklist-Irritability subscale (ABC-I),
            and

         2. moderate or higher on the Clinical Global Impressions-Severity scale;

    4. No changes in either medication or other interventions in the 4 weeks prior to
       randomization, and intention to remain on same dose for the duration of the study;

    5. Written informed consent from parent or legal guardian;

    6. Participant and family have the ability to comply with the protocol requirements, in
       the opinion of the investigator.

  Exclusion Criteria:

    1. Non-English speaking parents;

    2. Psychosis, bipolar disorder, major depressive disorder, obsessive compulsive disorder;

    3. Taking clobazam;

    4. Abnormal liver function tests: defined as ALT > twice ULN;

    5. Current use of medicinal cannabis, or use in the 4 weeks prior to screening;

    6. Pregnant or intending to become pregnant during the study, or breastfeeding;

    7. Known allergy to cannabidiol or cannabis products

Minimum age6 Years

Maximum age18 Years

GenderAll

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Other
Primary Sponsor Name: Murdoch Childrens Research Institute

Trial websitehttps://clinicaltrials.gov/show/NCT04821856

Trial IDNCT04821856








Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)

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Trial Information

Trial summary

A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone
therapy compared to placebo for the treatment of seizures in female children and young adults
with genetically confirmed PCDH19 gene mutation.

Broad Health Conditionrefractory seizures
epilepsy in children
seizure disorder

Specific Health ConditionPCDH19-Related Epilepsy

Trial FocusTreatment

Recruitment statusRecruiting

Phase of TrialPhase 3

Eligibility

Key inclusion criteria

Inclusion Criteria:

    -  Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant

    -  Failure to control seizures despite 2 or more anti-seizure medications

    -  12 seizures over a 12-week period of primary seizure types prior to screening

    -  On a stable regimen of anti-seizure treatments (Vagus nerve stimulator, ketogenic
       diet, and modified Atkins diet should be unchanged for 3 months prior to screening)

  Exclusion Criteria:

    -  Previous exposure to ganaxolone

    -  > 8 consecutive weeks of seizure freedom during the 12 weeks prior to screening

    -  Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted

    -  Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) is prohibited
       during the double-blind phase

    -  Exposure to any other investigational drug within 30 days or fewer than 5 half-lives
       prior to screening

Maximum age17 Years

GenderFemale

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Marinus Pharmaceuticals

Trial websitehttps://clinicaltrials.gov/show/NCT03865732

Trial IDNCT03865732








Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

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Trial Information

Trial summary

ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) that is developed as a clear gel
that can be applied to the skin (called transdermal delivery).

The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms.

Only participants from the ZYN2-CL-016 study who meet the inclusion criteria and none of the
exclusion criteria for study ZYN2-CL-017 are eligible.

Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.

Broad Health Condition

Specific Health ConditionFragile X Syndrome

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW,QLD,VIC,

Hospital
Westmead Children's Hospital

Hospital
Lady Cilento Children's Hospital - South Brisbane

Phase of TrialPhase 2/Phase 3

Eligibility

Key inclusion criteria

Inclusion Criteria:

    -  Participated in the ZYN2-CL-016 study.

    -  Patients and parents/caregivers agree to abide by all study restrictions and comply
       with all study procedures.

    -  Patients and parents/caregivers must be adequately informed of the nature, risks of
       the study, and give written informed consent prior to enrollment in ZYN2-CL-017.

    -  In the Investigator's opinion, the patients and parents/caregivers are reliable and
       are willing and able to comply with all protocol requirements and procedures.

    -  Females of childbearing potential must have a negative pregnancy test at all
       designated visits

  Exclusion Criteria:

    -  Patient is receiving any investigational drugs (not ZYN002) or using any experimental
       devices.

    -  Patient has an ongoing serious adverse event (SAE) or has experienced a SAE in
       ZYN2-CL-016, which in the opinion of the Investigator, should exclude them from
       participation.

    -  Females who are pregnant, nursing, or planning a pregnancy; females of childbearing
       potential and male patients with a partner of childbearing potential who are unwilling
       or unable to use an acceptable method of contraception for the duration of therapy and
       for three months after the last dose of trial drug.

    -  Patients who have alanine aminotransferase (ALT), aspartate aminotransferase (AST), or
       total bilirubin levels >= 2 times the upper limit of normal (ULN) or has alkaline
       phosphatase levels >= 3 times the ULN as determined from patient safety laboratories.

Minimum age3 Years

Maximum age18 Years

GenderAll

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Commercial sector/Industry
Primary Sponsor Name: Zynerba Pharmaceuticals, Inc.

Trial websitehttps://clinicaltrials.gov/show/NCT03802799

Trial IDNCT03802799