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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GTA (Glyceryl triacetate) | Dietary Supplement | 0.5 gr/kg x2/day syrup two times a day with increasing dose each 3 days till max 5gr /kg each day for 6 months |
| |
| GTA glyceryl triacetate | Drug | 0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg |
| Measure | Description | Time Frame |
|---|---|---|
| brain MRI at the end of the study | 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Gheona Alterescu, MD | Contact | 972-2-6666435 | gheona@szmc.org.il |
| Name | Affiliation | Role |
|---|---|---|
| Gheona Alterescu, M.D. | Shaare Zedek Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shaare Zedek Medical Center | Recruiting | Jerusalem | 91031 | Israel |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21474353 | Derived | Segel R, Anikster Y, Zevin S, Steinberg A, Gahl WA, Fisher D, Staretz-Chacham O, Zimran A, Altarescu G. A safety trial of high dose glyceryl triacetate for Canavan disease. Mol Genet Metab. 2011 Jul;103(3):203-6. doi: 10.1016/j.ymgme.2011.03.012. Epub 2011 Mar 15. |
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| ID | Term |
|---|---|
| D017825 | Canavan Disease |
| ID | Term |
|---|---|
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| D014215 | Triacetin |
| ID | Term |
|---|---|
| D014280 | Triglycerides |
| D005989 | Glycerides |
| D008055 | Lipids |
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| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |