Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Not Applicable
- Conditions
- Canavan Disease
- Registration Number
- NCT00724802
- Lead Sponsor
- Shaare Zedek Medical Center
- Brief Summary
Canavan disease is caused by Aspartoacylase deficiency. There is no treatment for the disease, but there is a food additive that includes acetate . We suggest an early treatment with acetate and a neurologic evaluation, including MRI, after 4 months of treatment. In any case the treatment will be stopped at the age of 22 months, when myelinization is ended.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 1
Inclusion Criteria
- clinical diagnosis of Canavan disease
Exclusion Criteria
- Age above 18 months
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method brain MRI at the end of the study 6 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Shaare Zedek Medical Center
🇮🇱Jerusalem, Israel