OTC DEFICIENCY

Clinical trials for OTC DEFICIENCY explained in plain language.

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Not yet finished but already full! 1 Terminated 1

One-Shot gene therapy aims to fix rare metabolic disorder

Disease control Ongoing

This study is testing whether a single dose of an experimental gene therapy called DTX301 can help the body control ammonia levels in people with late-onset OTC deficiency. About 32 participants, aged 12 and older, will be randomly assigned to receive either the gene therapy or a…

Matched conditions: OTC DEFICIENCY

Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control

Last updated Mar 18, 2026 14:40 UTC

OTC DEFICIENCY

Get alerted when new OTC DEFICIENCY trials appear

One-Shot gene therapy aims to fix rare metabolic disorder