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GTA-Glyceryltriacetate for Canavan Disease

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2006 by Sheba Medical Center.
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Sheba Medical Center
ClinicalTrials.gov Identifier:
NCT00278707
First received: January 15, 2006
Last updated: August 11, 2006
Last verified: August 2006
History of Changes

January 15, 2006
August 11, 2006
January 2006
Not Provided
  • 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
Same as current
Complete list of historical versions of study NCT00278707 on ClinicalTrials.gov Archive Site
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GTA-Glyceryltriacetate for Canavan Disease
Phase 1 Treatment With GTA in Two Infant With Canavan Disease

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

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).
Interventional
Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Infantile Canavan Disease
  • Deficiency Disease, Aspartoacylase
Drug: GTA: Glyceryltriacetate
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
5
July 2006
Not Provided

Inclusion Criteria:

  • Age below 15 months
  • Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

  • None
Both
up to 15 Months
No
Contact information is only displayed when the study is recruiting subjects
Israel
 
NCT00278707
SHEBA-05-3968-YA-CTIL
Not Provided
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Sheba Medical Center
Not Provided
Principal Investigator: Yair Anikster, MD PI Director Metabolic Disease Unit
Sheba Medical Center
August 2006

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