GTA-Glyceryltriacetate for Canavan Disease - Full Text View

Sponsored by:	Sheba Medical Center
Information provided by:	Sheba Medical Center
ClinicalTrials.gov Identifier:	NCT00278707

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 I

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Phase 1 Treatment With GTA in Two Infant With Canavan Disease

Resource links provided by NLM:

Genetics Home Reference related topics: Canavan disease leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts

MedlinePlus related topics: Leukodystrophies

U.S. FDA Resources

Further study details as provided by Sheba Medical Center:

Primary Outcome Measures:

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

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).

Eligibility

Ages Eligible for Study:	up to 15 Months
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Age below 15 months
Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

None

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00278707

Locations

Israel
Dr. Y. Anikster
Tel Aviv, Israel, 52621

Sponsors and Collaborators

Sheba Medical Center

Investigators

Principal Investigator:

Yair Anikster, MD PI

Director Metabolic Disease Unit

More Information

Additional Information:

Related Info This link exits the ClinicalTrials.gov site

Publications:

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.

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.

Study ID Numbers:	SHEBA-05-3968-YA-CTIL
Study First Received:	January 15, 2006
Last Updated:	August 11, 2006
ClinicalTrials.gov Identifier:	NCT00278707 History of Changes
Health Authority:	Israel: Israeli Health Ministry Pharmaceutical Administration

Keywords provided by Sheba Medical Center:

Canavan Disease
Aspartoacylase Deficiency
NAA
Acetate
Glyceryltriacetate

Study placed in the following topic categories:

Canavan Disease
Heredodegenerative Disorders, Nervous System
Malnutrition
Genetic Diseases, Inborn

Demyelinating Diseases
Nutrition Disorders
Neurodegenerative Diseases
Deficiency Diseases

Additional relevant MeSH terms:

Canavan Disease
Heredodegenerative Disorders, Nervous System
Malnutrition
Genetic Diseases, Inborn
Demyelinating Diseases

Nervous System Diseases
Nutrition Disorders
Neurodegenerative Diseases
Hereditary Central Nervous System Demyelinating Diseases
Deficiency Diseases

ClinicalTrials.gov processed this record on August 30, 2009