AADC Deficiency clinical trials at UCSF
1 research study open to eligible people
AADC deficiency is a rare inherited disease that lowers an enzyme needed for brain signals. UCSF is testing a viral gene therapy to deliver the enzyme directly into the brain. UCSF is tracking safety, dosing, and changes in movement in child participants.
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Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
open to eligible people ages 24 months and up
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.
San Francisco, California and other locations
Our lead scientists for AADC Deficiency research studies include Nalin Gupta, MD.
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