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

Brief 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.

Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.

Trial details

status

RECRUITING

phase

PHASE1, PHASE2

study type

INTERVENTIONAL

enrollment

7

sponsor

Rebecca Ahrens-Nicklas

conditions

Urea Cycle Disorders; Carbamoyl-Phosphate Synthase I Deficiency

interventions

BIOLOGICAL: LNP.UCD.ABE

start date

2026-08-07

start date type

ACTUAL

primary completion date

2028-09-01

has results

false

data source

ClinicalTrials.gov

data processed

2026-10-29T09:00:05

modifications

selected fields only; whitespace normalized; brief summary truncated to 2,000 characters