ANZCTR search results

This initial Phase 1 study will be conducted in normal healthy volunteers to gain an unconfounded understanding of the safety, tolerability, pharmacokinetics and pharmacodynamics of NJA-730. NJA-730 is being developed as an immunosuppressive drug for prevention and treatment of acute graft versus host disease associated with allogeneic hematopoietic stem cell transplant. This first-in-human, randomized, double-blind study will evaluate single ascending doses (SAD) and subsequently multiple ascending doses (MAD) of NJA-730/placebo. In the SAD part of the study, there will be 7 cohorts of 8 subjects each, with subjects in each cohort randomized in a 3:1 ratio to receive a single blinded dose of study drug i.e. NJA 730 or placebo, respectively (i.e. 6 subjects to receive NJA-730 and 2 subjects to receive placebo). In the MAD part of the study, there will be 3 cohorts of 8 subjects each with subjects in each cohort randomized in a 3:1 ratio to receive multiple blinded doses of NJA-730 or placebo, respectively, 3 times a week for a 1-week period (total 3 doses). A third part, an Extension Study (Part 3), will include approximately 24 healthy subjects divided into 4 cohorts of 6 subjects each. Subjects in each cohort will be randomized in a 2:1 ratio to receive a single blinded dose of study drug i.e. NJA-730 or placebo, respectively (i.e. 4 subjects to receive NJA-730 and 2 subjects to receive placebo).

Engaging patients in their own recovery after surgery is gaining momentum. A pilot study revealed that participants that received real-time recovery feedback (as a method of patient engagement) had better overall recovery at six weeks after surgery. Health-care providers must also kept informed of each individual patient’s recovery journey, as this alerts them to the patient with suboptimal recovery at the time that it is occurring, which may ultimately improve patient recovery in addition to what occurs when patients alone are informed. We will conduct a pilot trial investigating whether keeping both patients and their treating teams updated as to each patient’s recovery improve ultimate patient outcome. Recovery will be measured by the Postoperative Quality of Recovery Score (PostopQRS). Patients will be randomised to either receive no real-time feedback on their recovery assessment, or for there to be feedback to both patient and their treating health care team. This trial will determine the feasibility of providing both patients and their health teams with recovery data at the time of assessment, and will form the basis of a larger trial assessing the impact of each on the quality of a patient’s recovery journey.

To assess how long memantine HCl (50 mg) modified release prototype capsules stay in the stomach, as determined by imaging assessments (MRI or abdominal ultrasound) To evaluate the safety of memantine HCl modified release capsule formulations

As an emerging epidemic of cardiovascular disease, increasing numbers of patients are utilizing electrical cardioversion (CV) for treatment of symptomatic persistent atrial fibrillation (PeAF). The timing of CV following AF recurrence is dictated by a combination of factors, including patient symptoms, physician preference and resource availability. In addition to adverse effects on quality of life from prolonged AF duration, progressive adverse electrical and structural changes occur in the atria at different time points following arrhythmia onset . The clinical implications of delayed CV for intermittent PeAF are not well categorized, although some studies suggest these patients are at higher risk of AF recurrence . Due to barriers to accessing early elective cardioversion, including time taken to see a family physician, obtain specialist referral and wait for a scheduled CV, we adopted a policy of instructing patients to present directly to the Emergency department for early cardioversion. We sought to retrospectively compare a strategy of early ‘Emergency’ CV versus delayed ‘Elective’ CV for treatment of intermittent PeAF. We hypothesized that benefits of early CV may extend beyond symptoms, including prevention of adverse remodelling, reduction in recurrence risk and potentially lower utilization of AF ablation. In this observational retrospective cohort study, we plan to evaluate 150 patients presenting with symptomatic PeAF presenting to two centers in metropolitan Melbourne between 2/2014 – 7/2017. All included patients have a history of persistent AF, as defined by a previous or current episode of AF lasting longer than 7 days. We seek to compare two patient groups – those treated with Emergency vs Elective cardioversion strategies and included 75 consecutive patients from each group. Follow-up is over 12 months. Follow up for 12 months following cardioversion includes 12-lead ECG at onset of symptoms and during outpatient review at 3 months post discharge and 6 – 12 monthly thereafter. Referral for AF ablation is routinely initiated for symptomatic AF despite 1 – 2 antiarrhythmic agents. The primary endpoint is time to persistent AF recurrence. Secondary endpoints include AF duration prior to CV, changes in left atrial (LA) size on echocardiography from baseline to follow-up, modified European Heart Rhythm Association (EHRA) score at 12 months and time to referral for AF ablation. Medical records, including specialist and family physician visits, emergency and inpatient discharge summaries and echocardiographic data will be reviewed for recurrences, subsequent referrals for AF ablation and other endpoints.

This study is a Phase 1, randomised, dose escalation, double-blind, placebo-controlled study of cannabidiol (CBD) in healthy volunteers. The study is designed to evaluate the safety, tolerability and pharmacokinetics (PK) of a single dose of Cannabidiol in healthy volunteers. A total of 24 subjects will be enrolled in this study. Eight subjects in each cohort will be randomised to receive either CBD or placebo in a 3:1 ratio, so that 6 subjects receive CBD and 2 subjects receive placebo: Cohort 1: 5mg/kg Cohort 2: 10mg/kg Cohort 3: 20mg/kg. Sentinel dosing will be implemented in the first 2 subjects (1 CBD and 1 placebo) of Cohort 1; the rest of the subjects in the cohort will then be dosed if there are no significant safety concerns identified in the sentinel participants within at least the first 24 hours after administration of the oral dose (CBD or placebo). Sentinel dosing may be implemented for subsequent cohorts if deemed appropriate by the Safety Review Committee (SRC). The study will be conducted at a single study centre. Each participant will be involved in the study for a maximum of 15 days (screening period (up to 28 days); treatment period (3 days); follow-up visits (Days 4, 6 and 8) and telephone contact (Day 15). The SRC will undertake a dose-escalation review of all available safety data up to Day 8, approximately 2 weeks after the final subject in a cohort has been dosed and prior to subjects commencing the next dose. The primary objective of the study is to: • Assess the safety and tolerability of CBD following a single oral dose in healthy volunteers The secondary objective of the study is to: • Assess the PK of CBD following a single oral dose in healthy volunteers The primary endpoints to be evaluated in the study are: • Safety and tolerability of CBD, including review of: - adverse events - vital signs - 12-lead ECG - clinical laboratory assessments - concomitant medications. The secondary endpoints to be evaluated in the study are: • The PK of CBD, plasma concentration of CBD and PK parameters including: - AUClast – area under the plasma concentration versus time curve from time zero to the last quantifiable concentration - AUCinf – area under the plasma concentration versus time curve extrapolated to infinite time - Cmax – maximum observed plasma concentration - Tmax – time of maximum observed plasma concentration - Kel – apparent terminal elimination rate constant - T1/2 – apparent terminal elimination half-life The CBD to be manufactured for this study will be prepared as an oil for oral administration. Dosing will be based on subject body weight and the assigned dose cohort. The matching placebo will be composed of the same excipients as the study drug (without the CBD).

The aim of the study is to identify how women cope with pelvic girdle pain in pregnancy. Many women complain of pelvic girdle pain in pregnancy and seek information about this condition. Currently there is not much available information about this disorder, particularly in Australian women, and this study aims to address this lack of knowledge. Previous studies have so far found that women need to change their daily activities in order to try and cope. A qualitative design will be used to provide a rich description of the lived experience of women living with pelvic girdle pain in pregnancy via interviews, diary or focus groups.

The purpose of this study is determine whether exercise-based rehabilitation is effective in cancer survivors with Chemotherapy Induced Peripheral Neuropathy (CIPN). Who is it for? You may be eligible for this study if you are an adult with chemotherapy-induced peripheral neuropathy symptoms which persist after the completion of your chemotherapy treatment. Study details Participants in this study will be randomly sorted into two groups: - Group 1: participates in 3 weekly exercise sessions for 8 weeks. These sessions will be held face to face and either one-one-one or in small groups. Exercises will be individualized based on your balance abilities and strength and fitness levels and include cardiovascular, resistance, balance and stretching exercises. - Group 2: will receive an at-home exercise intervention after an 8 week delayed start. The home-exercises will also be completed 3 times a week for 8 weeks, be individualized, and include the same types of exercises as Group 1. Participants will also be required to complete fitness, nerve, functional and questionnaire assessments before and after their 8-week exercise program. It is hoped that this study will provide evidence regarding the effectiveness of exercise-based rehabilitation for people with chemotherapy induced peripheral neuropathy and establish the merits of home-based v clinic-based treatment.

Radiotherapy treats cancer using high energy x-rays in order to destroy cancer cells including prostate cancer via a linear accelerator machine. A new technology, known as the SUPER-I is a prospective study that will assess the safety and efficacy of using the ‘SeedTracker’ system in prostate radiotherapy in South Western Sydney Local Health District. Who is it for? You may be eligible for this study if you are an adult male receiving radiotherapy treatment for prostate cancer within the Liverpool or Macarthur cancer therapy centres. Study details All participants in this study will undergo their usual radiotherapy treatment schedule. As part of this study, during the radiotherapy treatment, participants will have radiotherapy markers implanted in the prostate in order to detect the position of the prostate in real-time. All blood tests will be performed as standard care by your specialist. It is hoped that this study will improve radiotherapy treatment delivery accuracy for those with prostate cancer.

Anxiety affects 7% of young people & only 50% receive help. This project aims to test the feasibility of a new, stepped care, internet based Cognitive behavioural treatment (iCBT) model for youth anxiety utilising the BRAVE Program. Therapist supported iCBT demonstrates equivalent efficacy with face to face CBT, but cost and insufficient numbers of clinicians means therapist guided iCBT is often not available or unsuitable to reach large numbers of anxious youth. Self help iCBT (no therapist) offers one solution. A national trial of BRAVE Self Help (>16,000 registrations in 2 years) showed meaningful anxiety reductions for youth completing the program. Many children failed to complete the treatment without support and not all were successfully treated. There is a need to identify models of care that can reach large numbers and provide appropriate support; such as a Stepped Care Model (SCM). Within a SCM, all young people would first receive the less intensive treatment. Those who have not responded well to the first step then receive a more intensive intervention. In this project, all participants would first receive 5 sessions of self help iCBT (step 1; psychoeducation, skills acquisition). Those who respond will receive 5 more self help sessions (skill rehearsal & maintenance). Those who fail to respond after step 1 would `step up' to receive their remaining 5 iCBT sessions with therapist guidance (step 2). Pilot evidence supports the use of a SCM in high intensity face to face CBT for childhood anxiety. To date, no studies of SCMs for iCBT exist. If a SCM approach, using self help plus therapist guided iCBT is as effective as therapist guided iCBT alone, we will have identified a scalable, easily disseminated model that can produce clinical outcomes similar to standard clinical therapy. This particular project utilises small doses of 'high intensity' (e.g. face-to-face) contact to supplement an evidence based treatment. Thus it provides a potential model of care that can first capitalize on the benefits of online interventions, and subsequently provide high intensity care, only when needed. This may have the capacity to effectively personalize the treatment to the young person. The study will also provide insights into the acceptability of face-to-face contact via videoconferencing in such contexts.

Hereditary Angiodema (HAE) is a rare disease caused by low levels of C1 serine protease inhibitor in the body. Deficiencies in this protein leads to increased activation of inflammatory pathways which can cause swelling, severe abdominal pain and airway obstruction that can be life threatening. ATN-249 is a potential once a day oral treatment for HAE. The purpose of this research study is to test the safety and tolerability of extended release formulations of ATN-249 as well as the pharmacokinetics and pharmacodynamics of the study drug. The study is open to healthy male volunteers and the research goals are: - Does the drug have any side-effects and is it well tolerated when given as a multiple dose over several days? - How much of the drug gets into the blood stream, and how long does the body take to get rid of it? This study will also look at how the human body uses ATN-249 at different dose levels and under different dosing regimens (single day dosing or 14-day dosing).