Overview

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

Status:
Active, not recruiting

Trial end date:
2024-01-01

Target enrollment:

Participant gender:

Summary

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.

Phase:

Phase 1

Details

Lead Sponsor:

Krystof Bankiewicz
Krzysztof Bankiewicz

Collaborators:

National Institute of Neurological Disorders and Stroke (NINDS)
National Institutes of Health (NIH)
University of California, San Francisco

Treatments:

Dopa Decarboxylase