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Sponsored by: |
Sheba Medical Center |
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Information provided by: | Sheba Medical Center |
ClinicalTrials.gov Identifier: | NCT00278707 |
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
Condition | Intervention | Phase |
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Infantile Canavan Disease Deficiency Disease, Aspartoacylase |
Drug: GTA: Glyceryltriacetate |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Phase 1 Treatment With GTA in Two Infant With Canavan Disease |
Estimated Enrollment: | 5 |
Study Start Date: | January 2006 |
Estimated Study Completion Date: | July 2006 |
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.
Ages Eligible for Study: | up to 15 Months |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Study ID Numbers: | SHEBA-05-3968-YA-CTIL |
Study First Received: | January 15, 2006 |
Last Updated: | August 11, 2006 |
ClinicalTrials.gov Identifier: | NCT00278707 |
Health Authority: | Israel: Israeli Health Ministry Pharmaceutical Administration |
Canavan Disease Aspartoacylase Deficiency NAA Acetate Glyceryltriacetate |
Canavan Disease Canavan disease Heredodegenerative Disorders, Nervous System Malnutrition Genetic Diseases, Inborn |
Demyelinating Diseases Nutrition Disorders Demyelinating diseases Neurodegenerative Diseases Deficiency Diseases |
Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases |