Clinical Trial

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

Recruiting Phase 1/2
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Summary
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.
Protocol Amendment History 3 changes
notable Primary completion pushed: 2028-07 -> 2028-09 2026-08-12
minor Completion pushed: 2028-07 -> 2028-09 2026-08-12
critical Recruitment opened 2026-08-08
Trial Details
NCT Number NCT07667387
Lead Sponsor Rebecca Ahrens-Nicklas
Conditions Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Enrollment 7 participants
Start Date 2026-08-07
Primary Completion 2028-09 (estimated)
Study Completion 2028-09 (estimated)
Updated on ClinicalTrials.gov 2026-08-11