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Inborn Urea Cycle Disorders clinical trials recruiting in the U.S.
8 studies for Inborn Urea Cycle Disorders are recruiting participants at sites across the United States.
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Recently updated studies
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
RecruitingObservational study
7 Months – 15 Months · All sexes · Updated Aug 31, 2026 · NCT06805695Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders
RecruitingPhase 1/2
24 Hours – 5 Years · All sexes · Updated Aug 26, 2026 · NCT07667387An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
RecruitingPhase 3
24 Hours – 7 Months · Men · Updated Aug 12, 2026 · NCT06255782Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task
RecruitingObservational studyHealthy volunteers welcome
Ages 16 to 40 · All sexes · Updated Jul 31, 2026 · NCT07701694At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism
Recruiting
Ages 12 and older · All sexes · Updated May 27, 2026 · NCT06953505Hepatic Histopathology in Urea Cycle Disorders
RecruitingObservational study
All ages · All sexes · Updated Jul 20, 2025 · NCT04908319Orphan Europe Carbaglu® Surveillance Protocol
RecruitingObservational study
All ages · All sexes · Updated Feb 7, 2024 · NCT03409003Systemic Biomarkers of Brain Injury From Hyperammonemia
RecruitingObservational study
Ages 7 to 18 · All sexes · Updated Feb 7, 2024 · NCT04602325From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.