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AAV2-hAADC Gene Therapy Expansion for AADC Deficiency
NCT02926066Phase 2Completed
- Sponsor
- National Taiwan University Hospital
- Start
- Nov 2016
- Primary completion
- Jan 2022
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of eladocagene exuparvovec in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by National Taiwan University Hospital. Primary endpoints: Evaluation of therapeutic effect (Peabody Developmental Motor Scales-2) at 13 months and Cerebrospinal fluid homovanillic acid levels at 13 months. Started Nov 2016; status: completed.
02
Drugs
eladocagene exuparvovec
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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