Clinical trials
Urea Cycle Disorders clinical trials
Below are recruiting urea cycle disorders clinical trials, each written for real people, not researchers. We’re tracking 11 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07667387RecruitingPhase 1/Phase 2
Base editing study for severe urea cycle disorders
This trial tests a new medicine called LNP.UCD.ABE that aims to fix a specific genetic change causing a severe urea cycle disorder. It uses a technique called base editing to correct the gene mutation, which may help your body break down ammonia better.
Philadelphia, PennsylvaniaAges 1 day–5 years - NCT06255782RecruitingPhase 3
Gene therapy study for baby boys with severe OTC deficiency
This trial tests a gene therapy called ECUR-506 for baby boys under 9 months old with a severe form of OTC deficiency. The goal is to see if it can help their bodies process ammonia better, reducing the need for a strict protein-restricted diet and medications.
Los Angeles, CaliforniaAges 1 day–7 months - NCT07701694Recruiting
Driving study for people with urea cycle disorders
This study evaluates how people with urea cycle disorders perform during a driving task. It may help researchers understand if the condition affects driving ability.
Memphis, TennesseeAges 16–40 - NCT04908319Recruiting
Studying liver tissue in urea cycle disorders
This study looks at liver tissue (under a microscope) in people with urea cycle disorders to better understand liver changes from the condition. It may help clinicians learn what to watch for and how to manage liver health, especially around liver biopsy or transplant.
Washington D.C., District of ColumbiaAges Any age - NCT05671666Recruiting
Testing urea processing in healthy people and urea cycle disorder
This study measures how your body breaks down protein-related waste and makes urea. It includes both healthy volunteers and people with urea cycle disorders, which may help researchers better understand the condition and improve testing.
Zurich, Canton of ZurichAges Any age - NCT06904027Recruiting
Glycerol phenylbutyrate for children with urea cycle disorders
This trial tests a medicine called glycerol phenylbutyrate in children with urea cycle disorders, which are genetic conditions that make it hard for the body to get rid of extra ammonia. The goal is to see if the medicine can help lower ammonia levels and manage the condition safely.
Beijing, Beijing MunicipalityAges birth–18 years - NCT00237315Recruiting
Long-term study of urea cycle disorders in affected families
This study follows people with urea cycle disorders (and some people who are very likely to have one) over time. It aims to better understand how these rare enzyme problems affect health, using medical tests and family information.
Los Angeles, CaliforniaAges Any age - NCT02935283Recruiting
MRI brain and thinking tests in urea cycle disorders
This study uses MRI scans and memory/thinking tests to understand brain effects of urea cycle disorders, like ornithine transcarbamylase deficiency (OTCD) and argininosuccinate (ASSD/ASLD). It may help researchers understand how brain function changes during and between metabolic crises.
Washington D.C., District of ColumbiaAges 7–50 - NCT06953505Recruiting
At-home ammonia monitoring for inherited metabolic disorders
This trial tests a device for measuring ammonia levels at home in people with rare metabolic conditions that cause high ammonia. It aims to make monitoring easier and more convenient for patients.
Portland, OregonAges 12 years+ - NCT04602325Recruiting
Blood and brain injury biomarker study for inherited metabolic disorders
This trial studies blood and other measurable signs (biomarkers) that may reflect brain injury after certain inherited metabolic conditions. Results may help doctors better detect and understand brain effects from these illnesses, especially episodes that affect the brain.
Washington D.C., District of ColumbiaAges 7–18 - NCT06805695Recruiting
Long-term follow-up for iECURE trial participants
This study follows up with people who took part in an earlier iECURE trial (such as a gene therapy study) to check on their health over time. It collects information to see how safe and lasting the treatment effects are.
LondonAges 7 months–1.3 years
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Urea Cycle Disorders trials by city
Studies with a site in or near these metro areas.
Urea Cycle Disorders trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for urea cycle disorders?
- Yes. Clin2 currently lists 11 recruiting urea cycle disorders studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a urea cycle disorders trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a urea cycle disorders trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.