Overview

This trial is active, not recruiting.

Conditions infantile canavan disease, deficiency disease, aspartoacylase
Treatment gta: glyceryltriacetate
Phase phase 1
Sponsor Sheba Medical Center
Start date January 2006
End date July 2006
Trial size 5 participants
Trial identifier NCT00278707, SHEBA-05-3968-YA-CTIL

Summary

The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment

Primary Outcomes

Measure
All primary outcome will be evaluated 4 months following the initiation of treatment:
time frame:
Neurological Status
time frame:
Brain Imaging: MRI & MRS
time frame:
NAA Levels in Urine
time frame:
Ophthalmologic Examination
time frame:

Eligibility Criteria

Male or female participants up to 15 months old.

Inclusion Criteria: - Age below 15 months - Biochemically diagnosed with Canavan Disease Exclusion Criteria: - None

Additional Information

Official title Phase 1 Treatment With GTA in Two Infant With Canavan Disease
Principal investigator Yair Anikster, MD PI
Description 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. 1. Two patients, aged less than 15 months, will receive daily doses of oral GTA 2. 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. 3. GTA has not been shown to cause any known toxicity, according to the Cosmetic Ingredient Review Expert Panel (Fiume, 2003).
Trial information was received from ClinicalTrials.gov and was last updated in August 2006.
Information provided to ClinicalTrials.gov by Sheba Medical Center.