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AAV2-hAADC Gene Therapy in Pediatric Patients With AADC Deficiency
NCT02852213Phase 1Recruiting
- Start
- Jul 2016
- Primary completion
- Aug 2027
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of ezaladcigene resoparvovec in Aromatic L-amino acid decarboxylase deficiency (AADC). Primary endpoints: Adverse events related to surgery and gene transfer at 2 years and Cerebrospinal fluid neurotransmitter metabolite concentrations at 1 year. Started Jul 2016; status: recruiting.
02
Drugs
ezaladcigene resoparvovec
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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