Clinical Trial

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

Study acronym: AADC
Recruiting Phase 1
View on ClinicalTrials.gov →
Summary
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.
Protocol Amendment History 17 amendments
This ClinicalTrials.gov record has been amended 17 times since 2016-07-28; most recent amendment 2026-07-09.
Status change: Active, Not Recruiting → Recruiting 2022-01-14
Status change: Recruiting → Active, Not Recruiting 2021-10-18
Trial Details
NCT Number NCT02852213
Lead Sponsor Krzysztof Bankiewicz
Collaborators: National Institute of Neurological Disorders and Stroke (NINDS), University of California, San Francisco
Conditions AADC Deficiency
Enrollment 42 participants
Start Date 2016-07-01
Primary Completion 2029-03 (estimated)
Study Completion 2037-03 (estimated)
Updated on ClinicalTrials.gov 2026-07-13