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NCT00278707 Clinical Trial

GTA-Glyceryltriacetate for Canavan Disease

Deficiency Disease, Aspartoacylase Clinical Trial

Official title:
Phase 1 Treatment With GTA in Two Infant With Canavan Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT00278707
Other study ID # SHEBA-05-3968-YA-CTIL
Secondary ID
Status Active, not recruiting
Phase Phase 1
First received January 15, 2006
Last updated August 11, 2006
Start date January 2006
Est. completion date July 2006

Study information
Verified date August 2006
Source Sheba Medical Center
Contact n/a
Is FDA regulated No
Health authority Israel: Israeli Health Ministry Pharmaceutical Administration
Study type Interventional

Clinical Trial Summary

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

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).

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 5
Est. completion date July 2006
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A to 15 Months
Eligibility Inclusion Criteria:

- Age below 15 months

- Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Drug:
• GTA: Glyceryltriacetate

Locations
Country Name City State
Israel Dr. Y. Anikster Tel Aviv

Sponsors (1)
Lead Sponsor Collaborator
Sheba Medical Center

Country where clinical trial is conducted

Israel, 

References & Publications (2)

Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA. Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. Proc Natl Acad Sci U S A. 2005 Apr 5;102(14):5221-6. Epub 2005 Mar 22. — View Citation

Mathew R, Arun P, Madhavarao CN, Moffett JR, Namboodiri MA. Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. J Pharmacol Exp Ther. 2005 Oct;315(1):297-303. Epub 2005 Jul 7. — View Citation

Outcome
Type Measure Description Time frame Safety issue
Primary All primary outcome will be evaluated 4 months following the initiation of treatment:
Primary Neurological Status
Primary Brain Imaging: MRI & MRS
Primary NAA Levels in Urine
Primary Ophthalmologic Examination

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