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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Tracking Information | ||||
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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 |
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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.
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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 |
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Condition ICMJE |
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Intervention ICMJE | Drug: GTA: Glyceryltriacetate | |||
Study Arms ICMJE | Not Provided | |||
Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Unknown status | |||
Enrollment ICMJE |
5 | |||
Original Enrollment ICMJE | Same as current | |||
Study Completion Date ICMJE | July 2006 | |||
Primary Completion Date | Not Provided | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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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 |
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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 |