AADC Deficiency clinical trials at UCSF
3 in progress, 1 open to eligible people
AADC deficiency affects children and limits their ability to make important brain chemicals. UCSF is conducting a clinical trial for pediatric patients. The goal is to determine safety and effectiveness of a new treatment. Two more trials are in progress, but not open to new patients.
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
open to eligible people ages 4 years 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
VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
Sorry, in progress, not accepting new patients
The objectives of this study are to assess the efficacy, safety and tolerability of VY-AADC02 in Patients with Parkinson's Disease with Motor Fluctuations.
San Francisco, California and other locations
Observational, Long-term Extension Study for Participants of Prior VY-AADC01 Studies
Sorry, in progress, not accepting new patients
An extension study for participants who have completed a prior VY-AADC01 clinical study
San Francisco, California and other locations
Our lead scientists for AADC Deficiency research studies include Nalin Gupta, MD Krystof Bankiewicz, MD.
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