GTA-Glyceryltriacetate for Canavan Disease - Tabular View

Tracking Information

First Received Date ^ICMJE

January 15, 2006

Last Updated Date

August 11, 2006

Start Date ^ICMJE

January 2006

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

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

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00278707 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

GTA-Glyceryltriacetate for Canavan Disease

Official Title ^ICMJE

Phase 1 Treatment With GTA in Two Infant With Canavan Disease

Brief Summary

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

Detailed 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.

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 Phase

Phase I

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Infantile Canavan Disease
Deficiency Disease, Aspartoacylase

Intervention ^ICMJE

Drug: GTA: Glyceryltriacetate

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Enrollment ^ICMJE

Completion Date

July 2006

Primary Completion Date

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Age below 15 months
Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

None

Gender

Both

Ages

up to 15 Months

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

Israel

Administrative Information

NCT ID ^ICMJE

NCT00278707

Responsible Party

Study ID Numbers ^ICMJE

SHEBA-05-3968-YA-CTIL

Study Sponsor ^ICMJE

Sheba Medical Center

Collaborators ^ICMJE

Investigators ^ICMJE

Principal Investigator:

Yair Anikster, MD PI

Director Metabolic Disease Unit

Information Provided By

Sheba Medical Center

Verification Date

August 2006

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP