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DTX301-CL301 Late-onset OTC Deficiency Gene Therapy Trial
2024-514337-38-00Phase 3Authorised
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2024
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 13
01
Overview
Phase 3, randomised, double-blind trial in ornithine transcarbamylase deficiency comparing Avalotcagene ontaparvovec with Placebo (2 arms). Primary endpoints: Plasma ammonia (AUC0-24) at Week 36 and Complete Responder rate at final study visit after DTX301 exposure. Planned start Sep 2024; status: authorised.
02
Drugs
avalotcagene ontaparvovec
03
Conditions
ornithine transcarbamylase deficiency
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