Ornithine Transcarbamylase Deficiency clinical trials at University of California Health
1 research study open to eligible people
Showing trials for
Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency
open to eligible people ages 12 years and up
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.