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Keyword: behcet
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ACTNSWNTQLDSATASVICWA

A Single Centre Phase II Study Of Haematopoietic Stem Cell Transplantation (HSCT) for Severe Auto-Immune Diseases.

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Trial Information

Trial summary

Diseases such as Scleroderma, Multiple Sclerosis (MS), Systemic Lupus Erythematosus (SLE), vasculitis, Chrohn’s disease, Behcet’s disease refractory to all available therapies and in the opinion of the referring physician, HSCT is a valid therapeutic option for that patient. These patients will require an independent physician to assess the patient’s suitability for HSCT. Patients will undergo GSCF stimulated stem cell collection following chemotherapy. Patients are readmitted for autlogous transplant and will have specific  high dose immunosuppresssive therapy depending on their disorder followed by  stem cell re infusion 

Broad Health ConditionAuto Immune disorders

Specific Health ConditionInflammatory and Immune System
Other inflammatory or immune system disorders
Musculoskeletal
Other muscular and skeletal disorders
Neurological
Multiple sclerosis

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW

Hospital
St Vincent's Hospital (Darlinghurst) - Darlinghurst

Postcode
2010 - Darlinghurst

Anticipated date of first participant enrolment8/03/2011

Anticipated date of last participant enrolment10/02/2021

Phase of TrialPhase 2

Has the study received ethics approval?Further information iconApproved

Eligibility

Key inclusion criteria

Adequate organ function as measured by: 
Cardiac LV Ejection Fraction greater than 45%, total Lung Capacity greater than 60%, Pulmonary artery pressure greater than 50mmHg, DLCO greater than or equal to 50%.
Negative serology for HBV, HCV and HIV.
Negative pregnancy test.
Able to provide informed consent and the absence of mental and cognitive deficits which can interfere with the capability of providing the informed consent.
Absence of severe chronic infection.
Severe auto-immune disease less than 7 years duration (excluding Multiple Sclerosis) OR Multiple sclerosis of any duration unresponsive to multiple standard therapies including corticosteroids.
HSCT deemed best high-intensity immunotherapeutic treatment in the opinion of the referring physician.

Sperm collection or ova cryopreservation is to be offered prior to HSCT in those of child-bearing age

Specific Inclusion Criteria for each disease:
1.Systemic Sclerosis: 
Early, rapidly progressive inflammatory diffuse scleroderma with truncal skin involvement and/or involving lungs and/or heart (myocarditis) which has failed to stabilize with anti-rheumatic agents. All patients with severe Systemic Sclerosis will undergo right catheterisation prior to cyclophosphamide mobilisation to assess for pulmonary artery pressure.  Ideally patients will also have signs of ongoing disease-related inflammation immediately prior to ASCT. Early disease is defined as disease in which the timing from second symptom onset to ASCT was 7 years or less

2. Systemic lupus erythematosus (SLE): 
Diagnosis according to ACR-criteria with antinuclear antibodies positive on at least two successive tests at three months interval plus disease duration less than 7 years since the diagnosis or first time of intensive immunosuppressive drugs. Unresponsive to multiple therapies including at least 6 months of the best standard local therapy using prednisone, intravenous cyclophosphamide or mycophenolate mofetil either alone or successively with or without anti CD 20 (Rituximab). Major organ damage (eg: cerebritis, nephritis, pulmonary, cardiac or skin vasculitis) is present despite optimal immunosuppression. 

3. Multiple Sclerosis (MS):
I. Diagnosis of relapsing remitting MS (RRMS) made by a Neurologist according to
the 2010 revised McDonald’s criteria
II EDSS score 0-6.5  NB: EDSS of 6.5 (this corresponds to able to walk, needing at most bilateral assistance to walk 20m without resting). Patients with an EDSS of 0-2 will require a second independent physician to assess the patient’s suitability for HSCT
III Disease duration of at least 15 years from diagnosis of MS
IV New MRI activity within last 12 months: Inflammatory active MS as defined by at least 1 Gd+ (>3mm) lesion (off steroids for one month) or at least 2 new T2 lesions on MRI within the last 12 months, compared to a reference scan not older than 36 months and preferably within the last 24 months from the date of eligibility review OR 
a history of highly active disease, as determined by previous MRI prior to commencement of high efficacy treatment (alemtuzumab and/or natalizumab), in patients where the long term immunosuppresive risk on treatment is determined to be of significance to patients morbidity/mortality (long term risk of infection, malignancy or organ failure secondary to treatment). Confirmation regarding suitability for HSCT from an external neurologist will be required in this case.
V Relapsing-remitting MS (RRMS), who have failed at least one licensed disease modifying drug of high efficacy (currently including alemtuzumab and natalizumab) because of demonstrated lack of efficacy (as evident from relapse, MRI activity as above, or EDSS increase) after being on Disease Modifying Therapy (DMT) for at least 6 months OR
in RRMS patients where the long term immunosuppressive risk on above treatment is determined to be of significance to patients morbidity/mortality (long term risk of infection, malignancy or organ failure secondary to treatment).

4. Other Auto-Immune Conditions:
Diseases such as vasculitis, Chrohn’s disease, Behcet’s disease refractory to all available therapies and in the opinion of the referring physician, HSCT is a valid therapeutic option for that patient. These patients will require an independent physician to assess the patient’s suitability for HSCT. 

Minimum age18 Years

Maximum age65 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key exclusion criteria

- If specific auto immune disorders does not meet the individual eligibility criteria.
- Any patient on the study treatment arm deemed not suitable for transplant by HSCT specialist
- Any patient unable to understand the purpose and risks of the study
- Patients deemed by their treating neurologist to have entered phase of secondary progressive
Contact details and further information

Sponsor Primary Sponsor Type: Hospital
Primary Sponsor Name: St Vincent's Hospital, Sydney
Primary Sponsor Address: 390 Victoria St Darlinghurst NSW 2010
Primary Sponsor Country: Australia

Trial IDACTRN12613000339752

Contact person for information and recruitmentDr
John Moore
St Vincent's Hospital, Sydney 390 Victoria St Darlinghurst NSW 2010
61 2 9355 5656
61 2 9355 5735
Further information iconsvhcancer_research@stvincents.com.au
Australia

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

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Trial Information

Trial summary

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux
Falls, South Dakota. It provides researchers with a centralized, international patient
registry for all rare diseases. This program allows patients and researchers to connect as
easily as possible to help advance treatments and cures for rare diseases. The CoRDS team
works with patient advocacy groups, individuals and researchers to help in the advancement of
research in over 7,000 rare diseases. The registry is free for patients to enroll and
researchers to access. Visit sanfordresearch.org/CoRDS to enroll.

Broad Health ConditionRare Diseases
Neglected Diseases
Orphan Diseases
Rare Disease Research
Registries
WAGR Syndrome
Ataxia
Cornelia de Lange Syndrome
Stickler Syndrome
Ataxia Telangiectasia
Kawasaki Disease
Batten Disease
Mucolipidosis IV
Klippel-Feil Syndrome
Multiple Endocrine Neoplasia
Atypical Hemolytic Uremic Syndrome
Undiagnosed
Uncommon Disease
Kabuki Syndrome
Hypersomnia
Hyperacusis
Kleine-Levin Syndrome
Marinesco-Sjogren Syndrome
Leiomyosarcoma
4p-/Wolf-Hirschhorn Syndrome
Hypophosphatasia
Narcolepsy
Wiedermann-Steiner Syndrome
Breast Implant-Associated Anaplastic Large Cell Lymphoma
Autoimmune/inflammatory Syndrome Induced by Adjuvants (ASIA)
Hemophagocytic Lymphohistiocytosis (HLH)
Behcet's Disease
Alagille Syndrome
Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia (IBMPFD)
Lowe Syndrome
Pitt Hopkins Syndrome
1p36 deletion syndrome
Jansen metaphyseal chondrodysplasia
Cockayne Syndrome
Chronic recurrent multifocal osteomyelitis (CRMO)
Malan syndrome
Hereditary Sensory and Autonomic Neuropathy

Specific Health ConditionRare Disorders
Undiagnosed Disorders
Disorders of Unknown Prevalence
Cornelia De Lange Syndrome
Prenatal Benign Hypophosphatasia
Perinatal Lethal Hypophosphatasia
Odontohypophosphatasia
Adult Hypophosphatasia
Childhood-onset Hypophosphatasia
Infantile Hypophosphatasia
Hypophosphatasia
Kabuki Syndrome
Bohring-Opitz Syndrome
Narcolepsy Without Cataplexy
Narcolepsy-cataplexy
Hypersomnolence Disorder
Idiopathic Hypersomnia Without Long Sleep Time
Idiopathic Hypersomnia With Long Sleep Time
Idiopathic Hypersomnia
Kleine-Levin Syndrome
Kawasaki Disease
Leiomyosarcoma
Leiomyosarcoma of the Corpus Uteri
Leiomyosarcoma of the Cervix Uteri
Leiomyosarcoma of Small Intestine
Acquired Myasthenia Gravis
Addison Disease
Hyperacusis (Hyperacousis)
Juvenile Myasthenia Gravis
Transient Neonatal Myasthenia Gravis
Williams Syndrome
Lyme Disease
Myasthenia Gravis
Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome)
Isolated Klippel-Feil Syndrome
Frasier Syndrome
Denys-Drash Syndrome
Beckwith-Wiedemann Syndrome
Emanuel Syndrome
Isolated Aniridia
Axenfeld-Rieger Syndrome
Aniridia-intellectual Disability Syndrome
Aniridia - Renal Agenesis - Psychomotor Retardation
Aniridia - Ptosis - Intellectual Disability - Familial Obesity
Aniridia - Cerebellar Ataxia - Intellectual Disability
Aniridia - Absent Patella
Aniridia
Peters Anomaly - Cataract
Peters Anomaly
Potocki-Shaffer Syndrome
Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11
Silver-Russell Syndrome Due to Imprinting Defect of 11p15
Silver-Russell Syndrome Due to 11p15 Microduplication
Syndromic Aniridia
WAGR Syndrome
Wolf-Hirschhorn Syndrome
4p16.3 Microduplication Syndrome
4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome
Autosomal Recessive Stickler Syndrome
Stickler Syndrome Type 2
Stickler Syndrome Type 1
Stickler Syndrome
Mucolipidosis Type 4
X-linked Spinocerebellar Ataxia Type 4
X-linked Spinocerebellar Ataxia Type 3
X-linked Intellectual Disability - Ataxia - Apraxia
X-linked Progressive Cerebellar Ataxia
X-linked Non Progressive Cerebellar Ataxia
X-linked Cerebellar Ataxia
Vitamin B12 Deficiency Ataxia
Toxic Exposure Ataxia
Unclassified Autosomal Dominant Spinocerebellar Ataxia
Thyroid Antibody Ataxia
Sporadic Adult-onset Ataxia of Unknown Etiology
Spinocerebellar Ataxia With Oculomotor Anomaly
Spinocerebellar Ataxia With Epilepsy
Spinocerebellar Ataxia With Axonal Neuropathy Type 2
Spinocerebellar Ataxia Type 8
Spinocerebellar Ataxia Type 7
Spinocerebellar Ataxia Type 6
Spinocerebellar Ataxia Type 5
Spinocerebellar Ataxia Type 4
Spinocerebellar Ataxia Type 37
Spinocerebellar Ataxia Type 36
Spinocerebellar Ataxia Type 35
Spinocerebellar Ataxia Type 34
Spinocerebellar Ataxia Type 32
Spinocerebellar Ataxia Type 31
Spinocerebellar Ataxia Type 30
Spinocerebellar Ataxia Type 3
Spinocerebellar Ataxia Type 29
Spinocerebellar Ataxia Type 28
Spinocerebellar Ataxia Type 27
Spinocerebellar Ataxia Type 26
Spinocerebellar Ataxia Type 25
Spinocerebellar Ataxia Type 23
Spinocerebellar Ataxia Type 22
Spinocerebellar Ataxia Type 21
Spinocerebellar Ataxia Type 20
Spinocerebellar Ataxia Type 2
Spinocerebellar Ataxia Type 19/22
Spinocerebellar Ataxia Type 18
Spinocerebellar Ataxia Type 17
Spinocerebellar Ataxia Type 16
Spinocerebellar Ataxia Type 15/16
Spinocerebellar Ataxia Type 14
Spinocerebellar Ataxia Type 13
Spinocerebellar Ataxia Type 12
Spinocerebellar Ataxia Type 11
Spinocerebellar Ataxia Type 10
Spinocerebellar Ataxia Type 1 With Axonal Neuropathy
Spinocerebellar Ataxia Type 1
Spinocerebellar Ataxia - Unknown
Spinocerebellar Ataxia - Dysmorphism
Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature
Spasticity-ataxia-gait Anomalies Syndrome
Spastic Ataxia With Congenital Miosis
Spastic Ataxia - Corneal Dystrophy
Spastic Ataxia
Rare Hereditary Ataxia
Rare Ataxia
Recessive Mitochondrial Ataxia Syndrome
Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature
Posterior Column Ataxia - Retinitis Pigmentosa
Post-Stroke Ataxia
Post-Head Injury Ataxia
Post Vaccination Ataxia
Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract
Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus
Non-hereditary Degenerative Ataxia
Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity
Olivopontocerebellar Atrophy - Deafness
NARP Syndrome
Myoclonus - Cerebellar Ataxia - Deafness
Multiple System Atrophy, Parkinsonian Type
Multiple System Atrophy, Cerebellar Type
Multiple System Atrophy
Maternally-inherited Leigh Syndrome
Machado-Joseph Disease Type 3
Machado-Joseph Disease Type 2
Machado-Joseph Disease Type 1
Leigh Syndrome
Late-onset Ataxia With Dementia
Infection or Post Infection Ataxia
GAD Ataxia
Hereditary Episodic Ataxia
Gliadin/Gluten Ataxia
Friedreich Ataxia
Fragile X-associated Tremor/Ataxia Syndrome
Familial Paroxysmal Ataxia
Exposure to Medications Ataxia
Episodic Ataxia With Slurred Speech
Episodic Ataxia Unknown Type
Episodic Ataxia Type 7
Episodic Ataxia Type 6
Episodic Ataxia Type 5
Episodic Ataxia Type 4
Episodic Ataxia Type 3
Episodic Ataxia Type 1
Epilepsy and/or Ataxia With Myoclonus as Major Feature
Early-onset Spastic Ataxia-neuropathy Syndrome
Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity
Early-onset Cerebellar Ataxia With Retained Tendon Reflexes
Early-onset Ataxia With Dementia
Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia
Dilated Cardiomyopathy With Ataxia
Cataract - Ataxia - Deafness
Cerebellar Ataxia, Cayman Type
Cerebellar Ataxia With Peripheral Neuropathy
Cerebellar Ataxia - Hypogonadism
Cerebellar Ataxia - Ectodermal Dysplasia
Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss
Brain Tumor Ataxia
Brachydactyly - Nystagmus - Cerebellar Ataxia
Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia
Autosomal Recessive Syndromic Cerebellar Ataxia
Autosomal Recessive Spastic Ataxia With Leukoencephalopathy
Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria
Autosomal Recessive Spastic Ataxia
Autosomal Recessive Metabolic Cerebellar Ataxia
Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine
Autosomal Recessive Ataxia, Beauce Type
Autosomal Recessive Ataxia Due to Ubiquinone Deficiency
Autosomal Recessive Ataxia Due to PEX10 Deficiency
Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia
Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency
Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency
Autosomal Recessive Congenital Cerebellar Ataxia
Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome
Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency
Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency
Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency
Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome
Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity
Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency
Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect
Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion
Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation
Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness
Autosomal Recessive Cerebellar Ataxia
Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly
Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation
Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy
Autosomal Dominant Spastic Ataxia Type 1
Autosomal Dominant Spastic Ataxia
Autosomal Dominant Optic Atrophy
Ataxia-telangiectasia Variant
Ataxia-telangiectasia
Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy
Autosomal Dominant Cerebellar Ataxia Type 4
Autosomal Dominant Cerebellar Ataxia Type 3
Autosomal Dominant Cerebellar Ataxia Type 2
Autosomal Dominant Cerebellar Ataxia Type 1
Autosomal Dominant Cerebellar Ataxia
Ataxia-telangiectasia-like Disorder
Ataxia With Vitamin E Deficiency
Ataxia With Dementia
Ataxia - Oculomotor Apraxia Type 1
Ataxia - Other
Ataxia - Genetic Diagnosis - Unknown
Acquired Ataxia
Adult-onset Autosomal Recessive Cerebellar Ataxia
Alcohol Related Ataxia
Multiple Endocrine Neoplasia
Multiple Endocrine Neoplasia Type II
Multiple Endocrine Neoplasia Type 1
Multiple Endocrine Neoplasia Type 2
Multiple Endocrine Neoplasia, Type IV
Multiple Endocrine Neoplasia, Type 3
Multiple Endocrine Neoplasia (MEN) Syndrome
Multiple Endocrine Neoplasia Type 2B
Multiple Endocrine Neoplasia Type 2A
Atypical Hemolytic Uremic Syndrome
Atypical HUS
Wiedemann-Steiner Syndrome
Breast Implant-Associated Anaplastic Large Cell Lymphoma
Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA)
Hemophagocytic Lymphohistiocytosis
Behcet's Disease
Alagille Syndrome
Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD)
Lowe Syndrome
Pitt Hopkins Syndrome
1p36 Deletion Syndrome
Jansen Type Metaphyseal Chondrodysplasia
Cockayne Syndrome
Chronic Recurrent Multifocal Osteomyelitis
CRMO
Malan Syndrome
Hereditary Sensory and Autonomic Neuropathy Type Ie
VCP Disease
Hypnic Jerking
Sleep Myoclonus
Mollaret Meningitis
Recurrent Viral Meningitis
CRB1
Leber Congenital Amaurosis
Retinitis Pigmentosa
Rare Retinal Disorder
KCNMA1-Channelopathy
Primary Biliary Cirrhosis
ZMYND11
Transient Global Amnesia
Glycogen Storage Disease
Alstrom Syndrome
White Sutton Syndrome
DNM1
EIEE31
Myhre Syndrome
Recurrent Respiratory Papillomatosis
Laryngeal Papillomatosis
Tracheal Papillomatosis
Refsum Disease
Nicolaides Baraitser Syndrome
Leukodystrophy
Tango2
Cauda Equina Syndrome
Rare Gastrointestinal Disorders
Achalasia-Addisonian Syndrome
Achalasia Cardia
Achalasia Icrocephaly Syndrome
Anal Fistula
Congenital Sucrase-Isomaltase Deficiency
Eosinophilic Gastroenteritis
Idiopathic Gastroparesis
Hirschsprung Disease
Rare Inflammatory Bowel Disease
Intestinal Pseudo-Obstruction
Scleroderma
Short Bowel Syndrome
Sacral Agenesis
Sacral Agenesis Syndrome
Caudal Regression
Scheuermann Disease
SMC1A Loss of Function Epilepsy

Recruitment statusRecruiting

Eligibility

Key inclusion criteria

Inclusion Criteria:

    -  Diagnosis of a rare disease, a disease of unknown prevalence, undiagnosed or an
       unaffected carrier of a rare/uncommon disease

  Exclusion Criteria:

    -  Diagnosis of a disease which is not rare

GenderAll

Can Healthy volunteers participate?No

Contact details and further information

Sponsor Primary Sponsor Type: Other
Primary Sponsor Name: Sanford Health

Trial websitehttps://clinicaltrials.gov/show/NCT01793168

Trial IDNCT01793168