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AADC Deficiency

1

Review clinical trials related to AADC Deficiency. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 42
Trial details
Phase: Phase 1Age: 24+Biological sex: AllType: InterventionalSponsor: Krzysztof BankiewiczUpdated: Aug 27, 2026Locations: 3
Eligibility criteria

Definite diagnosis of AADC deficiency, confirmed by at least two of the followin... [+8]

Intracranial neoplasm or any structural brain abnormality or lesion (e.g., sever... [+6]

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