AADC Deficiency clinical trials at UCSF
2 in progress, 1 open to eligible people
AADC deficiency is a rare genetic disorder that affects the nervous system. UCSF is conducting a study to check the safety and effectiveness of a new treatment for children with this condition. This trial involves adjusting the dose of a specific gene therapy.
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
VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
Sorry, in progress, not accepting new patients
The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations.
San Francisco, California and other locations
Our lead scientists for AADC Deficiency research studies include Nalin Gupta, MD.
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