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GTA-Glyceryltriacetate for Canavan Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00278707
Recruitment Status : Unknown
Verified August 2006 by Sheba Medical Center.
Recruitment status was:  Active, not recruiting
First Posted : January 18, 2006
Last Update Posted : August 15, 2006
Sponsor:
Information provided by:
Sheba Medical Center

Tracking Information
First Submitted Date  ICMJE January 15, 2006
First Posted Date  ICMJE January 18, 2006
Last Update Posted Date August 15, 2006
Study Start Date  ICMJE January 2006
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: January 15, 2006)
  • 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
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
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.

  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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Infantile Canavan Disease
  • Deficiency Disease, Aspartoacylase
Intervention  ICMJE Drug: GTA: Glyceryltriacetate
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Enrollment  ICMJE
 (submitted: January 15, 2006)
5
Original Enrollment  ICMJE Same as current
Study Completion Date  ICMJE July 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Age below 15 months
  • Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

  • None
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 0 Years to 15 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00278707
Other Study ID Numbers  ICMJE SHEBA-05-3968-YA-CTIL
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Not Provided
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Sheba Medical Center
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Yair Anikster, MD PI Director Metabolic Disease Unit
PRS Account 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