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GTA-Glyceryltriacetate for Canavan Disease

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified August 2006 by Sheba Medical Center.
Recruitment status was:  Active, not recruiting
Information provided by:
Sheba Medical Center Identifier:
First received: January 15, 2006
Last updated: August 11, 2006
Last verified: August 2006
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 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1 Treatment With GTA in Two Infant With Canavan Disease

Resource links provided by NLM:

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.

  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).

Ages Eligible for Study:   up to 15 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age below 15 months
  • Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00278707

Dr. Y. Anikster
Tel Aviv, Israel, 52621
Sponsors and Collaborators
Sheba Medical Center
Principal Investigator: Yair Anikster, MD PI Director Metabolic Disease Unit
  More Information

Additional Information:
Publications: Identifier: NCT00278707     History of Changes
Other Study ID Numbers: SHEBA-05-3968-YA-CTIL
Study First Received: January 15, 2006
Last Updated: August 11, 2006

Keywords provided by Sheba Medical Center:
Canavan Disease
Aspartoacylase Deficiency

Additional relevant MeSH terms:
Deficiency Diseases
Canavan Disease
Nutrition Disorders
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases processed this record on September 18, 2017