Clinical trials
Ornithine Transcarbamylase Deficiency clinical trials
Below are recruiting ornithine transcarbamylase deficiency clinical trials, each written for real people, not researchers. We’re tracking 5 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05092685RecruitingPhase 1/Phase 2
Gene therapy trial for children with OTC ammonia build-up
This early-phase study tests an AAV gene therapy meant to reduce ammonia levels in children with ornithine transcarbamylase (OTC) deficiency. It’s designed to slow or stop ammonia-related harm, while also carefully monitoring safety for years.
LondonAges birth–16 years - NCT07773246RecruitingPhase 1/Phase 2
Study of KRRO-121 in healthy adults and people with urea cycle disorder
This study tests an investigational drug called KRRO-121. It is first tested in healthy volunteers to check safety, and then in patients with urea cycle disorder (a condition where the body can't remove ammonia properly) to see if it helps.
Melbourne, VictoriaAges 18–65 - NCT06488313RecruitingPhase 2
Study of ARCT-810 for people with OTC deficiency
This trial tests an experimental medicine called ARCT-810 for people with Ornithine Transcarbamylase (OTC) deficiency, a condition that affects the liver's ability to remove ammonia from the body. The study aims to see if ARCT-810 can help lower ammonia levels safely.
Chevy Chase, MarylandAges 12 years+ - 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.
Aurora, ColoradoAges 7 months–1.3 years - 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
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Common questions
- Are there clinical trials for ornithine transcarbamylase deficiency?
- Yes. Clin2 currently lists 5 recruiting ornithine transcarbamylase deficiency 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 ornithine transcarbamylase deficiency 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 ornithine transcarbamylase deficiency 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.