Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of rAAVOlig001-ASPA Gene Therapy for Treatment of Children with Typical Canavan Disease

Condition studied

Canavan Disease, Canavan disease

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.

Endpoint detail

Rate of change in myelin volume in the brain as measured by SyMRI through Month 12., Rate of change from baseline in Gross Motor Function Measure-88 total score, comparing FIH gene therapy–treated cohort to an external natural history cohort., Rate of change from baseline in Gross Motor Function Measure-88 specific domains, comparing FIH gene therapy–treated cohort to an external natural history cohort: - Lying/rolling domain - Sitting domain - Crawling domain - Standing domain - Walking domain, Rate of change from baseline in multivariate MSEL domains age-equivalent scores compared with an external natural history cohort. MSEL domains include: Visual Reception, Fine Motor, Receptive Language, Expressive Language, Gross Motor, Rate of change from baseline in univariate MSEL domains age-equivalent scores compared with an external natural history cohort: - MSEL Receptive Language domain - MSEL Expressive Language domain - MSEL Visual Reception domain - MSEL Fine Motor domain - MSEL - Gross Motor domain, Change from baseline to Month 6 in urine NAA concentrations, as measured by biochemical assays