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Phase 3 study of DTX301 gene therapy in late-onset OTC deficiency
NL-OMON59864Phase 3Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jan 2023
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 3, single-arm trial of avalotcagene ontaparvovec in ornithine transcarbamylase deficiency, sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: Plasma ammonia at Week 36 and Complete Responder rate. Started Jan 2023; status: active, not recruiting.
02
Drugs
avalotcagene ontaparvovec
03
Conditions
ornithine transcarbamylase deficiency
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