Ongoing Clinical Trials for Inborn Error of Metabolism
This article provides information about ongoing clinical trials investigating treatments for inborn errors of metabolism. Currently, there is 1 clinical trial being conducted across multiple European countries, focusing on the long-term safety of a cell-based therapy called HepaStem for patients with specific metabolic and liver conditions including urea cycle disorders, Crigler-Najjar syndrome, and fibroinflammatory liver diseases.
Clinical trial locations
- Belgium
- Bulgaria
- France
- Poland
- Spain
Long-Term Safety Study of HepaStem for Patients with Urea Cycle Disorders, Crigler-Najjar Syndrome, and Fibroinflammatory Liver Diseases
This trial is examining the long-term safety of HepaStem, a cell-based therapy designed to support liver function. HepaStem uses specially prepared liver-derived cells that aim to help repair and regenerate damaged liver tissue. The study is focused on patients with three specific types of conditions: urea cycle disorders, Crigler-Najjar syndrome, and fibroinflammatory liver diseases.
Who can participate: This study is open to patients who have already received at least one infusion of HepaStem in a previous clinical trial conducted by the sponsor. Both adults and children can participate, including those from vulnerable populations who may need special care. Participants must be able to provide written informed consent, or in the case of children, their parents or legal representatives must provide consent on their behalf. If a minor becomes an adult during the study, they will need to provide their own consent. Similarly, adults who initially cannot consent due to their medical condition must provide their own consent once they are able to do so.
Who cannot participate: Patients who have not previously received HepaStem in an earlier study are not eligible. The trial only includes patients who participated in previous studies run by Promethera Biosciences or Promethera Therapeutics and who have one of the three specific conditions mentioned: urea cycle disorders, Crigler-Najjar syndrome, or fibroinflammatory liver diseases.
What the trial involves: The main goal of this study is to monitor the long-term safety of HepaStem therapy. Participants will not receive any new treatments but will be regularly observed to check for any potential side effects or health changes over time. Researchers will document any significant health events, such as organ transplantation, development of tumors, or diseases that might arise from infections or reactivation of dormant viruses. This monitoring will continue until July 2028, ensuring that comprehensive safety data is collected over several years. The information gathered will help researchers understand whether HepaStem remains safe for long-term use in treating these liver-related conditions.
Investigational treatment: HepaStem is a cell therapy that involves using special cells derived from the liver. The therapy is delivered directly into the bloodstream through an infusion. These cells are designed to provide the liver with the necessary building blocks to aid in healing and improve its function. By providing healthy liver cells, HepaStem aims to support patients whose liver function is compromised due to their underlying metabolic or liver disease.
Summary
Currently, there is one clinical trial investigating treatments for inborn errors of metabolism, specifically focusing on the long-term safety monitoring of HepaStem therapy. This trial is being conducted across five European countries: Belgium, Bulgaria, France, Poland, and Spain. The study is unique in that it focuses on follow-up care rather than initial treatment, observing patients who have already received the therapy in previous trials. The research is particularly relevant for patients with urea cycle disorders, Crigler-Najjar syndrome, and fibroinflammatory liver diseases. The multi-country approach reflects a coordinated effort to gather comprehensive safety data over an extended period, with monitoring planned to continue until 2028.