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Phase I/II Study of Gene Therapy for AADC Deficiency
jRCT2033210641Phase 2Completed
- Sponsor
- Gene Therapy Research Institute
- Start
- Mar 2022
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ezaladcigene resoparvovec in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by Gene Therapy Research Institute. Primary endpoint: Primary tolerability and safety. Started Mar 2022; status: completed.
02
Drugs
ezaladcigene resoparvovec
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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