Ornithine carbamoyltransferase deficiency
MONDO:0010703Ornithine transcarbamylase deficiency (OTCD) is a disorder of urea cycle metabolism and ammonia detoxification characterized by either a severe, neonatal-onset disease found almost exclusively in males, or later-onset (partial) forms of the disease. Both present with episodes of hyperammonemia that can be fatal and which can lead to neurological complications.
Also known as: OCT deficiency, OTC deficiency, OTCD, ornithine carbamoyltransferase deficiency, ornithine carbamoyltransferase deficiency disease, ornithine transcarbamylase deficiency, ornithine transcarbamylase deficiency, hyperammonemia due to, valproate sensitivity
20 clinical trials for this condition and its sub-types, 16 tagged with Ornithine carbamoyltransferase deficiency itself.
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Can a new injection tame the toxic ammonia of urea cycle disorders?
Disease control Not yet recruitingThis early-stage trial is evaluating an experimental drug called KRRO-121, given as a shot under the skin, to see if it is safe and tolerable. The study first tests KRRO-121 in healthy adults, then in people with urea cycle disorders—a group of inherited conditions that impair th…
Phase 1/2 • Sponsor: Korro Bio, Inc. • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
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Robotic surgery breakthrough aims to slash chest pain after esophageal cancer surgery
Symptom relief Not yet recruitingThis early-stage trial tests a new robotic surgical method for esophageal cancer that avoids cuts between the ribs, which may cause less chest pain and faster recovery. About 31 adults with stage I to III esophageal cancer will be randomly assigned to either the new approach or s…
Phase 1 • Sponsor: Centre hospitalier de l'Université de Montréal (CHUM) • Aim: Symptom relief
Last updated Jun 27, 2026 11:02 UTC
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Diabetic eye patients at risk for extra retinal scarring?
Knowledge-focused Not yet recruitingThis study will look at 225 adults with diabetic retinopathy to find out how many also develop an epiretinal membrane — a thin, scar-like layer on the retina that can blur vision. Researchers will use eye scans to detect the membrane and check for related factors like age, diabet…
Sponsor: Assiut University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC