GTA-Glyceryltriacetate for Canavan Disease
Recruitment status was Active, not recruiting
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Purpose
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Infantile Canavan Disease Deficiency Disease, Aspartoacylase |
Drug: GTA: Glyceryltriacetate |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase 1 Treatment With GTA in Two Infant With Canavan Disease |
- 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
| Estimated Enrollment: | 5 |
| Study Start Date: | January 2006 |
| Estimated Study Completion Date: | July 2006 |
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).
Eligibility| Ages Eligible for Study: | up to 15 Months |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age below 15 months
- Biochemically diagnosed with Canavan Disease
Exclusion Criteria:
- None
Contacts and Locations More Information
Additional Information:
Publications:
| ClinicalTrials.gov Identifier: | NCT00278707 History of Changes |
| Other Study ID Numbers: | SHEBA-05-3968-YA-CTIL |
| Study First Received: | January 15, 2006 |
| Last Updated: | August 11, 2006 |
| Health Authority: | Israel: Israeli Health Ministry Pharmaceutical Administration |
Keywords provided by Sheba Medical Center:
|
Canavan Disease Aspartoacylase Deficiency NAA Acetate Glyceryltriacetate |
Additional relevant MeSH terms:
|
Deficiency Diseases Canavan Disease Malnutrition Nutrition Disorders Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases |
Nervous System Diseases Leukoencephalopathies Demyelinating Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Metabolism, Inborn Errors Metabolic Diseases |
ClinicalTrials.gov processed this record on May 16, 2013