Ornithine transcarbamylase deficiency
Clinical trials for Ornithine transcarbamylase deficiency explained in plain language.
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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: ORNITHINE TRANSCARBAMYLASE DEFICIENCY
Phase 3 • Sponsor: iECURE, Inc. • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC
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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: ORNITHINE TRANSCARBAMYLASE DEFICIENCY
Sponsor: iECURE, Inc. • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC