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

The company Ultragenyx Pharmaceutical Inc is enrolling patients into the clinical trial investigating Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency.

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.

The trial is designed to enroll male and female 12 Years and older. The study start date is May 2022.

This page provides a more detailed overview of this clinical trial: https://ichgcp.net/clinical-trials-registry/NCT05345171.