Clinical trial
Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE
NCT07667387 is registered in ClinicalTrials.gov with Rebecca Ahrens-Nicklas as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE
- Lead sponsor
- Rebecca Ahrens-Nicklas
- Phase
- Phase 1/2
- Overall status
- Recruiting
- Start date
- August 7, 2026
- Completion date
- September 30, 2028
- Enrollment
- 7 (estimated)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.
carbamoyl-phosphate synthase i deficiency, urea cycle disorders