OTC DEFICIENCY
Clinical trials for OTC DEFICIENCY explained in plain language.
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Gene therapy aims to tame deadly ammonia spikes in rare disease
Disease control OngoingThis study tests a one-time gene therapy (DTX301) for adults with late-onset OTC deficiency, a genetic disorder that causes dangerous ammonia buildup in the blood. The goal is to safely keep ammonia levels normal without long-term medication. About 32 participants will receive ei…
Matched conditions: OTC DEFICIENCY
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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New shot targets rare ammonia disorder in early human trial
Disease control OngoingThis early-stage study tests a new drug called CMP-CPS-001 in healthy adults and women with a genetic condition called OTC deficiency, which makes it hard for the body to remove ammonia. The main goal is to check safety and how the drug moves through the body. Participants receiv…
Matched conditions: OTC DEFICIENCY
Phase: PHASE1 • Sponsor: CAMP4 Therapeutics Corporation • Aim: Disease control
Last updated May 14, 2026 12:01 UTC