Status: Not yet recruiting
A Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders.
This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of LNP.UCD.ABE in 5 pediatric subjects with severe infantile-onset UCDs. This is a master clinical protocol in which subjects with a variant in a urea cycle disorder (UCD) gene (CPS1, OTC, ASS1, ASL, ARG, NAGS, or SLC25A15) that is demonstrated to be amenable to corrective editing by an adenine base editor (ABE) would be eligible for enrollment.
Participants needed: 7
Trial details
Phase: Phase 1, Phase 2Age: 24-5Biological sex: AllType: InterventionalSponsor: Rebecca Ahrens-NicklasUpdated: Jun 29, 2026Locations: 1
Eligibility criteria
Diagnosis of a severe urea cycle disorder, in the judgement of the investigators... [+7]
Abnormal liver function, electrolyte, coagulation, or blood count laboratory val... [+7]