Ongoing Clinical Trials for Urea Cycle Disorder
There are currently 2 ongoing clinical trials investigating treatments for Urea Cycle Disorder. These studies are taking place across several European countries including Belgium, Bulgaria, France, Netherlands, Poland, and Spain. The trials are testing different approaches: one involves a new injectable medication called CMP-CPS-001 specifically for patients with Ornithine Transcarbamylase (OTC) genetic variations, while the other is a long-term safety study of HepaStem, a cell therapy that has been given to patients in previous trials.
Clinical trial locations
- Belgium
- Bulgaria
- France
- Netherlands
- Poland
- Spain
Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders
This trial is testing an investigational medication called CMP-CPS-001 in people who have a specific genetic variation affecting the Ornithine Transcarbamylase enzyme, as well as in healthy volunteers. The medication is given as an injection under the skin.
Main focus: The primary goal of this study is to evaluate whether CMP-CPS-001 is safe and well-tolerated. Researchers will monitor participants’ health through various medical tests, blood samples, and urine samples to understand how the medication works in the body. The study will test different dose levels to find the most appropriate dose. Some participants will receive the actual medication while others will receive placebo. The study uses special compounds taken by mouth to help track how the body processes substances.
Who can participate: The study is looking for participants aged 18 to 65 years old for one part of the study, and aged 16 to 65 years for another part. Female participants in certain study parts must have confirmed heterozygous OTC genotype, meaning they have one copy of the specific genetic variation. Participants must have a Body Mass Index between 18.0 and 32.0, and weigh no more than 110 kilograms. All participants must sign a written consent form agreeing to take part.
Who cannot participate: People who have had a liver transplant or are currently on a transplant waiting list cannot join the study. Those with active cancer or a history of cancer within the past 5 years, severe kidney problems, or who are currently pregnant or breastfeeding are also excluded. People who have participated in another clinical trial within the past 30 days, have known allergies to similar medications, or have severe mental health conditions that could interfere with following the study procedures cannot participate. Those with serious or unstable medical conditions, blood disorders, or a history of drug or alcohol abuse within the past year are also excluded.
Investigational drug: CMP-CPS-001 is an experimental medication administered by injection under the skin. It is still in early research stages and is being tested to understand how safe it is for people with abnormal OTC genes. The medication is being developed to address the body’s difficulty in processing nitrogen-containing compounds, which is a key problem in urea cycle disorders.
Long-Term Safety Study of HepaStem for Patients with Urea Cycle Disorders, Crigler-Najjar Syndrome, and Fibroinflammatory Liver Diseases
This trial is focused on monitoring the long-term safety of a treatment called HepaStem. HepaStem is a type of cell therapy that uses specially prepared liver cells to help treat certain liver-related conditions.
Main focus: The purpose of this study is to monitor patients who have previously received at least one infusion of HepaStem in earlier clinical trials. Participants will not receive any new treatments in this study but will be regularly observed to check for any potential side effects or health changes over time. The study aims to document any significant health events, such as organ transplantation, development of tumors, or any disease linked to infections. This monitoring will help researchers understand the long-term effects of the therapy and ensure that it remains safe. The study is expected to continue until July 2028.
Who can participate: This study is specifically for patients who have already received at least one infusion of HepaStem during a previous clinical study. Participants must be able to understand and give written informed consent to participate. For children, consent must be given by parents or a legal representative, and if the child can understand the study, they should also agree. If a patient is underage when joining the study and becomes an adult during the study, they must provide their own consent as an adult. For adults who cannot give consent due to a condition affecting their ability to understand, a legal representative must provide consent, but the patient must give their own consent as soon as they are able. The study includes both male and female patients of various ages.
Who cannot participate: Patients who have not received at least one infusion of HepaStem in a previous clinical study cannot participate. Those who did not take part in a previous study conducted by Promethera Biosciences or Promethera Therapeutics are also excluded. Additionally, patients who do not have conditions such as urea cycle disorders, Crigler-Najjar syndrome, or fibroinflammatory liver diseases are not eligible for this study.
Investigational drug: HepaStem is a cell therapy that involves using special cells derived from the liver. These cells are designed to help repair and regenerate damaged liver tissue. The therapy is delivered directly into the bloodstream through an infusion. HepaStem aims to support liver function and improve the health of patients with liver conditions by providing the liver with healthy cells to aid in its healing process.
Summary
Currently, there are two active clinical trials for Urea Cycle Disorder taking place across Europe. The first trial, conducted in the Netherlands, is testing a new injectable medication called CMP-CPS-001 specifically for patients with OTC genetic variations. This is an early-phase study focused on understanding the safety and tolerability of the drug at different dose levels.
The second trial is much broader in scope and is being conducted across five countries: Belgium, Spain, France, Bulgaria, and Poland. This is a long-term safety monitoring study for patients who have previously received HepaStem, a liver cell therapy. Unlike the first trial, participants in this study will not receive new treatments but will be followed to assess the long-term safety of a therapy they have already received.
Both trials represent different approaches to treating the condition: one tests a completely new medication in its early stages, while the other monitors the long-term effects of an existing cell therapy. The geographic concentration of the HepaStem study across multiple European countries suggests a well-established research network for this particular therapy.
