GTA-Glyceryltriacetate for Canavan Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00278707
• Date of registration: 15/01/2006
• Prospective Registration: No
• Primary sponsor: Sheba Medical Center
• Public title: GTA-Glyceryltriacetate for Canavan Disease
• Scientific title: Phase 1 Treatment With GTA in Two Infant With Canavan Disease
• Date of first enrolment: January 2006
• Target sample size: 5
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT00278707
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Israel

Contacts

• Name: Yair Anikster, MD PI
• Affiliation: Director Metabolic Disease Unit

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age below 15 months

- Biochemically diagnosed with Canavan Disease

Exclusion Criteria:

- None



Age minimum: N/A
Age maximum: 15 Months
Gender: Both

Health Condition(s) or Problem(s) studied

Deficiency Disease, Aspartoacylase

Infantile Canavan Disease

Intervention(s)

Drug: GTA: Glyceryltriacetate

Primary Outcome(s)

NAA Levels in Urine

Ophthalmologic Examination

All primary outcome will be evaluated 4 months following the initiation of treatment:

Brain Imaging: MRI & MRS

Neurological Status

Secondary ID(s)

SHEBA-05-3968-YA-CTIL

Source(s) of Monetary Support

Please refer to primary and secondary sponsors