Urea cycle disorders, inborn
Clinical trials for Urea cycle disorders, inborn explained in plain language.
Never miss a new study
Get alerted when new Urea cycle disorders, inborn trials appear
Sign up with your email to follow new studies for Urea cycle disorders, inborn, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Gene therapy trial offers hope for babies with rare, deadly metabolic disease
Disease control Recruiting nowThis study tests a one-time gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic disorder that prevents the body from breaking down ammonia. The goal is to see if the treatment is safe and can reduce dangerous ammonia levels…
Matched conditions: UREA CYCLE DISORDERS, INBORN
Phase 3 • Sponsor: iECURE, Inc. • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC
-
Can a single gene therapy dose keep working for 14 years?
Knowledge-focused Recruiting nowThis study follows people who received an investigational gene therapy in an earlier iECURE trial for a urea cycle disorder, a condition that can cause dangerous ammonia buildup. Researchers will track safety, side effects, and whether the therapy's effects last over up to 14.5 y…
Matched conditions: UREA CYCLE DISORDERS, INBORN
Sponsor: iECURE, Inc. • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC