Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase 3 DTX301 Gene Therapy in Late-onset OTC Deficiency
jRCT2073220097Phase 3Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jun 2024
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
01
Overview
Phase 3, randomised, double-blind trial of avalotcagene ontaparvovec in ornithine transcarbamylase deficiency (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: Percentage of Participants at Week 64 Who Have Achieved Complete Response (DTX301 vs Placebo) and Plasma Ammonia 24-hour AUC0-24 at Week 64. Started Jun 2024; status: active, not recruiting.
02
Drugs
avalotcagene ontaparvovec
03
Conditions
ornithine transcarbamylase deficiency
Beyond the preview
Go deeper on jRCT2073220097.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on jRCT2073220097.