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LNP.UCD.ABE for Urea Cycle Disorders

This study is testing a new treatment called LNP.UCD.ABE for children with severe urea cycle disorders (UCDs). UCDs are genetic conditions where the body can't properly remove ammonia, a waste product, which can be very harmful. LNP.UCD.ABE is a personalized treatment that uses tiny fat particles (lipid nanoparticles) to deliver genetic material (mRNA) to correct the specific genetic problem causing the UCD. Researchers want to see if LNP.UCD.ABE is safe, how well people tolerate it, and if it helps improve their condition. This study is for children aged 24 hours to 5 years who have a severe UCD and a specific genetic mutation that LNP.UCD.ABE can target. The study aims to enroll 7 participants.

Study design
This is a single-site, open-label study, meaning everyone knows what treatment is being given. It is a Phase 1/2 study, which typically looks at safety and initial effectiveness, and plans to enroll 7 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety and tolerability for 52 weeks after receiving a single dose of LNP.UCD.ABE.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07667387

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

Recruiting
PHASE1Ages 24–5InterventionalTreatment
Rebecca Ahrens-Nicklas
~7 participants
Updated 2026-08-26 on ClinicalTrials.gov
What's tested:LNP.UCD.ABE

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety and tolerability of a single intravenous dose of LNP.UCD.ABE
Measured over 52 weeks

Conditions

Urea Cycle Disorders
Carbamoyl-Phosphate Synthase I Deficiency

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Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

  • Rebecca Ahrens-Nicklas, M.D., Ph.D. · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia

Who to contact

Sarah McCague
[email protected]
267-426-1464
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

If the patient is taking a nitrogen scavenger medication, their ammonia level may currently be in the normal range
If the patient is diagnosed prenatally, then personal history, family history, or analysis of mutations should indicate a high likelihood of a severe UCD. 6. Subjects more than 8 weeks from the initial diagnosis of a UCD must have demonstrated:
a persistent need for dietary protein restriction and chronic administration of a nitrogen scavenger medication, AND / OR
a recurrent hyperammonemic event AND / OR
a history of a hyperammonemia-induced seizure 7. Weight \>3.5 kg at the time of screening 8. Legal guardian(s) capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

What this trial measures

  • Safety and tolerability of a single intravenous dose of LNP.UCD.ABE52 weeks

    Incidence of treatment-emergent adverse events as assessed by CTCAE version 6.0 criteria at 52 weeks after LNP.UCD.ABE administration.