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GTA-Glyceryltriacetate for Canavan Disease

Primary Purpose

Infantile Canavan Disease, Deficiency Disease, Aspartoacylase

Status
Unknown status
Phase
Phase 1
Locations
Israel
Study Type
Interventional
Intervention
GTA: Glyceryltriacetate
Sponsored by
Sheba Medical Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Infantile Canavan Disease focused on measuring Canavan Disease, Aspartoacylase Deficiency, NAA, Acetate, Glyceryltriacetate

Eligibility Criteria

0 Years - 15 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Age below 15 months Biochemically diagnosed with Canavan Disease Exclusion Criteria: None

Sites / Locations

  • Dr. Y. Anikster

Outcomes

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

Secondary Outcome Measures

Full Information

First Posted
January 15, 2006
Last Updated
August 11, 2006
Sponsor
Sheba Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00278707
Brief Title
GTA-Glyceryltriacetate for Canavan Disease
Official Title
Phase 1 Treatment With GTA in Two Infant With Canavan Disease
Study Type
Interventional

2. Study Status

Record Verification Date
August 2006
Overall Recruitment Status
Unknown status
Study Start Date
January 2006 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
July 2006 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Sheba Medical Center

4. Oversight

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Infantile Canavan Disease, Deficiency Disease, Aspartoacylase
Keywords
Canavan Disease, Aspartoacylase Deficiency, NAA, Acetate, Glyceryltriacetate

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
5 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
GTA: Glyceryltriacetate
Primary Outcome Measure Information:
Title
All primary outcome will be evaluated 4 months following the initiation of treatment:
Title
Neurological Status
Title
Brain Imaging: MRI & MRS
Title
NAA Levels in Urine
Title
Ophthalmologic Examination

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
15 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age below 15 months Biochemically diagnosed with Canavan Disease Exclusion Criteria: None
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yair Anikster, MD PI
Organizational Affiliation
Director Metabolic Disease Unit
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dr. Y. Anikster
City
Tel Aviv
ZIP/Postal Code
52621
Country
Israel

12. IPD Sharing Statement

Citations:
PubMed Identifier
16002461
Citation
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. doi: 10.1124/jpet.105.087536. Epub 2005 Jul 7.
Results Reference
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PubMed Identifier
15784740
Citation
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. doi: 10.1073/pnas.0409184102. Epub 2005 Mar 22.
Results Reference
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GTA-Glyceryltriacetate for Canavan Disease

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