OTC DEFICIENCY
Clinical trials for OTC DEFICIENCY explained in plain language.
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Gene therapy aims to tame rare metabolic disorder
Disease control OngoingThis Phase 3 trial tests a gene therapy called DTX301 for people with late-onset OTC deficiency, a rare genetic disorder that causes dangerous ammonia buildup. The study involves 37 participants and compares the gene therapy to a placebo. The goal is to see if the treatment can h…
Matched conditions: OTC DEFICIENCY
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New shot targets rare liver disorder in early human tests
Disease control OngoingThis early-stage study tests a new drug called CMP-CPS-001 in healthy adults and women who carry a gene for OTC deficiency, a rare liver condition that can cause dangerous ammonia buildup. The main goal is to check the drug's safety and how the body processes it. Participants rec…
Matched conditions: OTC DEFICIENCY
Phase: PHASE1 • Sponsor: CAMP4 Therapeutics Corporation • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC