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GTA-Glyceryltriacetate for Canavan Disease

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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

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

Condition or disease Intervention/treatment Phase
Infantile Canavan Disease Deficiency Disease, Aspartoacylase Drug: GTA: Glyceryltriacetate Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 5 participants
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
Study Start Date : January 2006
Study Completion Date : July 2006

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Leukodystrophies

Arms and Interventions
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Outcome Measures
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Primary Outcome Measures :
  1. All primary outcome will be evaluated 4 months following the initiation of treatment:
  2. Neurological Status
  3. Brain Imaging: MRI & MRS
  4. NAA Levels in Urine
  5. Ophthalmologic Examination

Eligibility Criteria
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Ages Eligible for Study:   0 Years to 15 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age below 15 months
  • Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

  • None
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00278707


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

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ClinicalTrials.gov Identifier: NCT00278707    
Other Study ID Numbers: SHEBA-05-3968-YA-CTIL
First Posted: January 18, 2006    Key Record Dates
Last Update Posted: August 15, 2006
Last Verified: August 2006
Keywords provided by Sheba Medical Center:
Canavan Disease
Aspartoacylase Deficiency
NAA
Acetate
Glyceryltriacetate
Additional relevant MeSH terms:
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Canavan Disease
Deficiency Diseases
Malnutrition
Nutrition Disorders
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Leukoencephalopathies
Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases