Single-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency

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.


Krzysztof Bankiewicz

Status of enrollment

Accepting new patients

Ages Eligible for Study

For people ages 4 years and up

Disease indication

AADC deficiency

Principal Investigator

Nalin Gupta, MD


Shivani Mahuvakar

[email protected]


Additional study eligibility details can be found at UCSF Clinical Trials.