Condition

Ornithine Transcarbamylase Deficiency clinical trials and research

• 4Clinical trials
• 3Countries

Newest Ornithine Transcarbamylase Deficiency clinical trials

All recruiting trials (4)

• Long-term follow-up for participants in an iECURE protocol

  This study looks at the long-term safety and effectiveness of a treatment for people who have already been part of an earlier study, while also tracking the health of those who didn’t receive the treatment.

  Recruiting at 4 sites iECURE, Inc. · Aurora, Colorado and 3 other locations · NCT06805695

• Evaluating ARCT-810 for treating OTC deficiency

  This study is testing a new treatment called ARCT-810 to see if it can help adolescents and adults with OTC deficiency feel better while they continue their usual care.

  Recruiting Phase 2 Arcturus Therapeutics, Inc. · Chevy Chase, Maryland · NCT06488313

• Gene therapy for children with Ornithine Transcarbamylase deficiency

  This study is testing a new gene therapy for children with Ornithine Transcarbamylase deficiency to see if it can help their liver produce a missing enzyme and reduce dangerous ammonia levels in their blood.

  Recruiting Phase 1/2 University College, London · London · NCT05092685

• Evaluating liver tissue in patients with urea cycle disorders

  This study looks at liver tissue from people with urea cycle disorders to see how these conditions affect the liver, especially in those who have had a liver transplant or biopsy.

  Recruiting Baylor College of Medicine · Washington D.C., District of Columbia and 1 other locations · NCT04908319

Related topics

Quick answers

How many Ornithine Transcarbamylase Deficiency clinical trials are recruiting right now?

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

Where are these trials running?

In 3 countries. The most sites are in United States (3 trials), United Kingdom (2 trials), France (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 1, Phase 1/2: 1. 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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