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Phase 3 study of DTX301 gene therapy in late-onset ornithine transcarbamylase deficiency
2020-003384-25Phase 3Active
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Nov 2021
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in ornithine transcarbamylase deficiency comparing DTX301 with Placebo (2 arms). Primary endpoint: Response status at Week 64. Started Nov 2021; status: active.
02
Drugs
avalotcagene ontaparvovec
03
Conditions
ornithine transcarbamylase deficiency
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