AAV2-hAADC

DRUG3 trials

Sponsors

Krzysztof Bankiewicz, National Taiwan University Hospital

Conditions

AADC DeficiencyAromatic Amino Acid Decarboxylase DeficiencyEarly Onset Parkinson DiseaseParkinson's Disease (PD)

Phase 1

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

RecruitingNCT02852213

Krzysztof BankiewiczAADC Deficiency

Start: 2016-07-01End: 2037-08-01Target: 42Updated: 2026-08-27

AAV2-hAADC for Parkinson's Disease (PDCS-01)

Not yet recruitingNCT07267065

Krzysztof BankiewiczEarly Onset Parkinson Disease, Parkinson's Disease (PD)

Start: 2026-08-01End: 2028-12-01Target: 9Updated: 2026-08-27

Phase 2

A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

CompletedNCT02926066

National Taiwan University HospitalAromatic Amino Acid Decarboxylase Deficiency

Start: 2016-11-09End: 2022-05-16Updated: 2023-02-10

Related Papers

Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With <scp>Aromatic L‐Amino Acid Decarboxylase</scp> Deficiency Using an <scp>MR</scp> ‐Compatible Cannula: 48 Weeks of Follow‐Up

Journal of Inherited Metabolic Disease2026-02-22

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

Nature Communications2021-07-12159 citations