● Recruiting

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

Condition: AADC Deficiency · Sponsor: Krzysztof Bankiewicz

PhasePhase 1

Planned participants42

Who can joinAll sexes, 24 Months to no upper limit

Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Andrea Davis, MS · 614-688-6412 · andrea.davis@osumc.edu

Always discuss trial participation with your own doctor first.

Locations (3)

University of California San Francisco, Benioff Children's Hospital	San Francisco, California, United States	Recruiting
Nationwide Children's Hospital	Columbus, Ohio, United States	Recruiting
The Ohio State University Medical Center	Columbus, Ohio, United States	Recruiting

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Source record: clinicaltrials.gov/study/NCT02852213