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The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
The primary objective of the SAF001 study is the long-term safety surveillance of the patients post infusion with HepaStem. Furthermore, the evolution of both the metabolic condition and the quality of life are followed. As much as possible, the surveillance will mimic the standard follow-up of the respective diseases (standard of care). The surveillance will end when the patient is undergoing an organ transplant or takes part in another research study. This surveillance will last up to a maximum of 48 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Inborn errors of liver metabolism |
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| Measure | Description | Time Frame |
|---|---|---|
| Characterisation of the long term safety profile of HepaStem therapy. | Assessment of safety will be achieved by evaluating the following parameters
| 4 years |
| Measure | Description | Time Frame |
|---|---|---|
| To characterize the disease evolution after having received HepaStem therapy and to report on general safety. | This assessment is based on the evaluation of:
Indication I: Crigler-Najjar syndrome
Indication II: Urea cycle disorders
|
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Main Inclusion Criteria:
- Subject having received HepaStem during a former interventional clinical study and who have terminated their participation in that study.
Exclusion Criteria:
- Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.
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The SAF001 study will include all patients having received infusions of HepaStem in any former interventional study conducted by Promethera Biosciences.
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| Name | Affiliation | Role |
|---|---|---|
| Françoise Smets, MD | Cliniques universitaires Saint-Luc (Belgium) | Principal Investigator |
| Dries Dobbelaere, MD/Prof | CHRU de Lille - Hopital Jeanne de Flandre (France) | Principal Investigator |
| Isabel Gonçalves, MD/Prof | Hospital Pediátrico de Coimbra (Portugal) | Principal Investigator |
| Stephanie Grunewald, MD | Great Ormond Street Children Hospital | Principal Investigator |
| Giuliano Torre, MD | IRCCS OSPEDALE PEDIATRICO DEL BAMBINO GESÃ (Roma) | Principal Investigator |
| Hanna Mandel, MD | Rambam Health Corporation | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Promethera Biosciences | Mont-Saint-Guibert | 1435 | Belgium |
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| ID | Term |
|---|---|
| D056806 | Urea Cycle Disorders, Inborn |
| D003414 | Crigler-Najjar Syndrome |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| 4 years |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D006933 | Hyperbilirubinemia, Hereditary |