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AADC Deficiency clinical trials

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NCT ID: NCT02852213 Recruiting - AADC Deficiency Clinical Trials

A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

Start date: July 2016
Phase: Phase 1
Study type: Interventional

The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.