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

- 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

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Ages Eligible for Study: | 0 Years 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:
- None

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
Israel | |
Dr. Y. Anikster | |
Tel Aviv, Israel, 52621 |
Principal Investigator: | Yair Anikster, MD PI | Director Metabolic Disease Unit |
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 |
Canavan Disease Aspartoacylase Deficiency NAA Acetate Glyceryltriacetate |
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 |