Condition

Urea Cycle Disorders clinical trials and research

• 5Clinical trials
• 4Countries

Newest Urea Cycle Disorders clinical trials

All recruiting trials (5)

• KRRO-121 for hyperammonemia in urea cycle disorders: a first-in-human study

  This study tests KRRO-121, a treatment designed to help clear ammonia, in adults with urea cycle disorders and in healthy volunteers to see if it is safe and how it acts in the body.

  Recruiting Phase 1/2 Korro Bio, Inc. · Melbourne, Victoria · NCT07773246

• LNP.UCD.ABE for severe urea cycle disorders in young children

  This trial tests a single IV dose of LNP.UCD.ABE in five young children with severe urea cycle disorders to see if it is safe and may help reduce ammonia buildup.

  Recruiting Phase 1/2 Children's Hospital of Philadelphia · Philadelphia, Pennsylvania · NCT07667387

• Home Ammonia Monitoring for People with Metabolic Ammonia Disorders

  This study tests whether people with certain ammonia metabolism disorders can successfully measure their ammonia levels at home every day.

  Recruiting Sequitur Health Corp. · Portland, Oregon · NCT06953505

• Glycerol phenylbutyrate for Chinese children and teens with urea cycle disorders

  This project will try glycerol phenylbutyrate in children and teens in China who have urea cycle disorders to see if it lowers ammonia and reduces hyperammonemic crises over five years.

  Recruiting at 5 sites Tongji Hospital · Beijing, Beijing Municipality and 4 other locations · NCT06904027

• Analyzing urea production in healthy individuals and patients with urea cycle disorders

  This study is testing how well the body makes urea in healthy people and those with urea cycle disorders to better understand these conditions and improve treatments.

  Recruiting University Children's Hospital, Zurich · Zurich, Canton of Zurich · NCT05671666

Related topics

Quick answers

How many Urea Cycle Disorders clinical trials are recruiting right now?

5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 4 countries. The most sites are in United States (2 trials), Australia (1 trial), China (1 trial), Switzerland (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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