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drugset / Trial / NCT05345171

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171 ↗

Phase 3 Active not recruiting 37 enrolled Ultragenyx Pharmaceutical Inc
RandomizedCrossoverQuadruple-blindTreatment

Summary

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.

Timeline

Start
2022-10-18
Primary completion
2027-09
Completion
2031-03

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject AVALOTCAGENE ONTAPARVOVEC Gene therapy (AAV / viral vector) — Intravenous
Background Prednisolone Other / unclassified — —

Indications

ornithine carbamoyltransferase deficiency