| 1 |
NCT02851563 |
Recruiting |
A Natural History Study of Canavan Disease |
|
|
Observational |
|
- Massachusetts General Hospital
- Universitätsklinikum Hamburg-Eppendorf
- New York University School of Medicine
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in Gross Motor Function Measure over time
- Change in magnetic resonance imaging findings over time
|
10 |
All |
up to 99 Years (Child, Adult, Senior) |
NCT02851563 |
2015P002400 |
|
May 2016 |
May 2018 |
May 2018 |
August 1, 2016 |
May 16, 2017 |
|
- Massachusetts General Hospital
Boston, Massachusetts, United States - NYU Langone Medical Center
New York, New York, United States - University Medical Center Hamburg-Eppendorf
Hamburg, Germany
|
| 2 |
NCT00278707 |
Unknown † |
GTA-Glyceryltriacetate for Canavan Disease |
- Infantile Canavan Disease
- Deficiency Disease, Aspartoacylase
|
- Drug: GTA: Glyceryltriacetate
|
Interventional |
Phase 1 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- All primary outcome will be evaluated 4 months following the initiation of treatment:
- Neurological Status
- Brain Imaging: MRI & MRS
- (and 2 more...)
|
5 |
All |
up to 15 Months (Child) |
NCT00278707 |
SHEBA-05-3968-YA-CTIL |
|
January 2006 |
|
July 2006 |
January 18, 2006 |
August 15, 2006 |
|
- Dr. Y. Anikster
Tel Aviv, Israel
|
| 3 |
NCT00724802 |
Unknown † |
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan |
|
- Dietary Supplement: GTA (Glyceryl triacetate)
- Drug: GTA glyceryl triacetate
|
Interventional |
Not Applicable |
- Shaare Zedek Medical Center
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- brain MRI at the end of the study
|
1 |
All |
up to 18 Months (Child) |
NCT00724802 |
canavangta |
|
March 2008 |
August 2008 |
|
July 30, 2008 |
June 21, 2011 |
|
- Shaare Zedek Medical Center
Jerusalem, Israel
|
| 4 |
NCT00657748 |
Withdrawn |
Lithium and Acetate for Canavan Disease |
- Canavan Disease
- Infantile
- Deficiency Disease
- (and 2 more...)
|
- Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)
|
Interventional |
Phase 2 |
- Assistance Publique - Hôpitaux de Paris
- European Leukodystrophy Association
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary outcome will be a decrease of the NAA peak (> 20%) or the appearance of an acetate peak at the end of the treatment (10 months), using spectroscopy-MRI.
- Secondary outcomes will be assessed at 10 months (end of the treatment): -Improvement of neuromotor performances (GMFM and Mullen scales), spasticity, and neurological severity
- -Improvement of epilepsy (number of seizures)
- -Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine).
|
0 |
All |
1 Year to 15 Years (Child) |
NCT00657748 |
P070803 |
|
September 2009 |
September 2010 |
January 2011 |
April 14, 2008 |
April 21, 2015 |
|
|
| 5 |
NCT01999257 |
Completed |
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. |
- Tay Sachs Disease
- Canavan Disease
- Familial Dysautonomia
|
- Other: Online pre-test genetic education tool
|
Interventional |
Not Applicable |
- McGill University Health Center
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Health Services Research
|
- Knowledge of Ashkenazi Jewish genetic conditions
- Patient anxiety
- Satisfaction with web-based/in-person genetic counselling
- Perceived risk of having a child with an Ashkenazi Jewish genetic condition
|
60 |
All |
18 Years and older (Adult, Senior) |
NCT01999257 |
3281 |
|
July 2014 |
April 2015 |
August 2017 |
December 3, 2013 |
August 17, 2017 |
|
- Montreal General Hospital (MUHC)
Montreal, Quebec, Canada
|
| 6 |
NCT00654433 |
Terminated |
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases |
- Inherited Metabolic Diseases
- Lysosomal Storage Disorders
- Peroxisomal Storage Diseases
- (and 2 more...)
|
|
Interventional |
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases
- To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
- To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
|
40 |
All |
up to 16 Years (Child) |
NCT00654433 |
UCBT-002
BB-IND#13502 |
UCBT-002 |
March 2008 |
July 2011 |
November 2011 |
April 8, 2008 |
July 8, 2014 |
|
- Mattel Children's Hospital at UCLA
Los Angeles, California, United States - Mt. Sinai Medical Center
New York, New York, United States - Duke University
Durham, North Carolina, United States
|
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