{"id":"nct:NCT07667387","kind":"trial","title":"Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders","authors":["Rebecca Ahrens-Nicklas"],"author_count":1,"year":2026,"published":"2026-06-25","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT07667387","source_url":"https://clinicaltrials.gov/study/NCT07667387","archive_url":"https://space-kitty.com/papers/nct:NCT07667387","updated":"2026-08-26","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"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.","abstract_license":"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","license":"ClinicalTrials.gov terms (attribution, processing date, modifications noted)","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":["Urea Cycle Disorders","Carbamoyl-Phosphate Synthase I Deficiency"],"details":{"acronym":null,"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"},"provenance":"clinicaltrials","attribution":"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.","topics":[{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","domain_slug":"medicine"}],"cited_in_dossiers":[]}