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Phase 1 Treatment With GTA in Two Infant With Canavan Disease
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
Details
| Lead sponsor | Sheba Medical Center |
|---|---|
| Phase | Phase 1 |
| Status | UNKNOWN |
| Enrolment | 5 |
| Start date | 2006-01 |
| Completion | 2006-07 |
Conditions
- Infantile Canavan Disease
- Deficiency Disease, Aspartoacylase
Interventions
- GTA: Glyceryltriacetate
Primary outcomes
- 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
Countries
Israel