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

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

First listed

Jun 25, 2026

Start date

Aug 31, 2026

Status verified

Aug 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

• Sarah McCague
  267-426-1464
  [email protected]

Locations (1)

Find similar trials in Philadelphia, PA

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