Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency
Status:
Recruiting
Trial end date:
2028-12-01
Target enrollment:
Participant gender:
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 with removal of
dietary protein restriction and alternative pathway medication.