Clinical Trial

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

Name: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency — clinical trial record (NCT05345171)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Active, Not Recruiting Phase 3

View on ClinicalTrials.gov →

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.

Protocol Amendment History 1 change

notable Enrollment increased: 32 -> 37 participants 2026-06-03

Trial Details

NCT Number	NCT05345171
Lead Sponsor	Ultragenyx Pharmaceutical Inc
Conditions	OTC Deficiency
Enrollment	37 participants
Start Date	2022-10-18
Primary Completion	2027-09 (estimated)
Study Completion	2031-03 (estimated)
Updated on ClinicalTrials.gov	2026-06-02