A Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders.

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Rebecca Ahrens-Nicklas
Study ID
NCT07667387
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

  • Carbamoyl-Phosphate Synthase I Deficiency
  • Urea Cycle Disorders

Eligibility Criteria

Sex
ALL
Age
24 Hours - 5 Years
Healthy Volunteers
Not accepted

Interventions

  • LNP.UCD.ABE — BIOLOGICAL
    Each subject will have a personalized variant-specific LNP.UCD.ABE developed and evaluated in real time. Each member of the LNP.UCD.ABE drug product (DP) family is a lipid nanoparticle (LNP)-based editing therapeutic comprising lipid excipients, a messenger RNA (mRNA) drug substance (DS) encoding an adenine base editor (ABE), and a single guide RNA (gRNA) DS.

Study Details

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.

Key Dates

Start date
Jul 31, 2026
Status verified
Jun 2026
Primary completion
Jul 31, 2028
Completion
Jul 31, 2028

Study Design

Enrollment
7 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Experimental

Primary Outcome Measure

Safety and tolerability of a single intravenous dose of LNP.UCD.ABE [ Time Frame: 52 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

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By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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