GTA-Glyceryltriacetate for Canavan Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00278707|
Recruitment Status : Unknown
Verified August 2006 by Sheba Medical Center.
Recruitment status was: Active, not recruiting
First Posted : January 18, 2006
Last Update Posted : August 15, 2006
|Condition or disease||Intervention/treatment||Phase|
|Infantile Canavan Disease Deficiency Disease, Aspartoacylase||Drug: GTA: Glyceryltriacetate||Phase 1|
Canavan Disease is caused by a deficiency in the enzyme named Aspartoacylase (ASPA). This disease is a devastating, progressive disease with no available treatment. As a result of the ASPA deficiency, there are high levels of N-acetylaspartate (NAA) and low levels of L-aspartate and acetate.
We hypothesize that one of the functions of ASPA is to provide sufficient levels of acetate for CNS myelinization. For this reason, we offer to supplement acetate levels by the oral administration of glyceryl triacetate (GTA). Such treatment must be offered to patients before the age of 18 months, prior to the termination of CNS myelinization.
- Two patients, aged less than 15 months, will receive daily doses of oral GTA
- The daily dose will be increased incrementally until the maintenance dose is reached. This will be done under close monitoring of the patients, including periodic blood gas sampling.
- GTA has not been shown to cause any known toxicity, according to the Cosmetic Ingredient Review Expert Panel (Fiume, 2003).
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1 Treatment With GTA in Two Infant With Canavan Disease|
|Study Start Date :||January 2006|
|Study Completion Date :||July 2006|
- All primary outcome will be evaluated 4 months following the initiation of treatment:
- Neurological Status
- Brain Imaging: MRI & MRS
- NAA Levels in Urine
- Ophthalmologic Examination
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00278707
|Dr. Y. Anikster|
|Tel Aviv, Israel, 52621|
|Principal Investigator:||Yair Anikster, MD PI||Director Metabolic Disease Unit|