Date: 2014-10-15
Type of information: Initiation of preclinical development
phase: 2b-3
Announcement: authorization of the trial in Belgium
Company: Promethera Biosciences (Belgium)
Product: Promethera® HepaStem
Action
mechanism: Promethera® HepaStem is a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorder. The treatment is based on allogeneic adult liver stem cell technology. The major innovation of Promethera® HepaStem resides in the simplicity of the treatment - a simple injection into the vein leading to the liver – which may make it possible to avoid radical and invasive surgery such as a liver transplant. If this therapy proves to be safe and effective, as many as a hundred patients could be treated from a single liver, thus largely overcoming the organ shortage problem.
Disease: urea cycle disorders (UCD)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases - Liver diseases
Country:
Trial
details: HEP002 is a prospective, open label, multicenter efficacy and safety study with Promethera® HepaStem. The primary objective is to assess the efficacy of Promethera® HepaStem in patients with urea cycle deficiencies during the year following treatment.
Latest
news: * On October 15, 2014, Promethera Biosciences, a Belgian biotechnology company developing Promethera® HepaStem, a cell-based therapy for the treatment of both orphan liver-based metabolic diseases and acquired liver diseases, announced that Belgium has authorized the conduct of a trial designed to treat paediatric patients with urea cycle disorders (UCD) in a Phase IIb/III study (HEP002). This first agreement officially opens the start of the second clinical trial conducted by the company. Belgian patients will be recruited and treated in Cliniques Universitaires Saint-Luc in Brussels. In the first clinical trial HEP001 Promethera® HepaStem demonstrated to be a safe treatment that can result in functional metabolic improvement in UCD patients. As of today, all patients have been treated and are in the follow-up period. Observational study (OBS001): OBS001 is a retrospective and prospective observational study for paediatric patients suffering from Urea Cycle disorders or Crigler-Najjar syndrome. Patients are followed prospectively for a maximum of four years. This multicenter study is currently being conducted in Belgium and France. Long-term safety study (SAF001): This trial is proposed for patients who have completed a study with HepaStem. The main objective of the SAF001 is to document the safety of HepaStem for a period of four years. The SAF001 protocol has been approved in Belgium, France, Italy, United Kingdom, Israel and Portugal.
