Urea cycle disorder – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:18:26 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Urea cycle disorder – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders https://clinicaltrials.eu/trial/study-of-cmp-cps-001-injection-in-healthy-volunteers-and-patients-with-abnormal-ornithine-transcarbamylase-otc-genotype-for-treating-urea-cycle-disorders/ Wed, 29 Apr 2026 15:06:49 +0000 https://clinicaltrials.eu/trial/study-of-cmp-cps-001-injection-in-healthy-volunteers-and-patients-with-abnormal-ornithine-transcarbamylase-otc-genotype-for-treating-urea-cycle-disorders/ This clinical trial studies the investigational drug CMP-CPS-001 in people with Urea Cycle Disorders (UCD), specifically those with an abnormal Ornithine Transcarbamylase (OTC) genetic variation. The study includes both healthy volunteers and individuals with this genetic condition. The medication is given as an injection under the skin (subcutaneous injection).

The study uses several compounds to help track how the body processes substances: [13C]Sodium Acetate and 15NH4Cl, which are taken by mouth as solutions. The purpose of this research is to evaluate if CMP-CPS-001 is safe and well-tolerated when given to study participants. Some participants will receive the study medication while others will receive placebo.

During the study, participants will receive either single doses or multiple doses of the medication. The researchers will monitor participants’ health through various medical tests and collect blood and urine samples to understand how the medication works in the body. The study medication will be tested at different dose levels to find the most appropriate dose.

]]> Long-Term Safety Study of HepaStem for Patients with Urea Cycle Disorders, Crigler-Najjar Syndrome, and Fibroinflammatory Liver Diseases https://clinicaltrials.eu/trial/long-term-safety-study-of-hepastem-for-patients-with-urea-cycle-disorders-crigler-najjar-syndrome-and-fibroinflammatory-liver-diseases/ Wed, 29 Apr 2026 14:24:53 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-hepastem-for-patients-with-urea-cycle-disorders-crigler-najjar-syndrome-and-fibroinflammatory-liver-diseases/ This clinical trial is focused on understanding the long-term safety of a treatment called HepaStem. HepaStem is a type of cell therapy, which means it uses specially prepared cells to help treat certain diseases. The diseases being studied in this trial include urea cycle disorders, Crigler-Najjar syndrome, and various fibroinflammatory liver diseases. These conditions can affect how the liver functions and processes substances in the body.

The purpose of this study is to monitor patients who have previously received at least one infusion of HepaStem in earlier clinical trials. The study aims to ensure that the treatment remains safe over a long period. Patients who have participated in these earlier studies will continue to be observed to check for any potential side effects or health changes. This includes looking out for serious events such as organ transplants, the development of tumors, or any diseases that might arise from infections or reactivation of dormant viruses.

Participants in this study will not receive any new treatments but will be regularly monitored to gather information about their health following their initial HepaStem treatment. This monitoring will help researchers understand the long-term effects of the therapy and ensure that it is safe for continued use in treating these liver-related conditions.

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