RecruitingNCT05299710
Rare condition
Ornithine transcarbamylase deficiency
9
studies recruiting now
as of 7 Sept 2026
61
studies registered in total
as of 7 Sept 2026
4
countries with a recruiting site
as of 7 Sept 2026
3 Feb 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT06805695
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
RecruitingPhase 3NCT06255782
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
RecruitingNCT02132741
Optical Coherence Tomography And NEphropathy: The OCTANE Study
RecruitingNCT04908319
Hepatic Histopathology in Urea Cycle Disorders
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 9 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About Ornithine transcarbamylase deficiency
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the gene involved (OTC).
Treatments being studied
1 approved treatment and 2 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Sodium Phenylbutyrate (Ambutyrate)
Phase 3Avalotcagene Ontaparvovec
Phase 3Sodium Acetate (Sodium acetate component of procalamine)
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).