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Research line 'Rare anemias and bone marrow failure'
The research line 'rare anemias and bone marrow failure' is driven by both fundamental researchers in the Creveld-laboratory and clinical researchers in the Van Creveldkliniek. This offers us to apply this bench to bedside to a variety of diseases.
Research includes both studies that are initiated by our researchers and studies that are being performed on behalf of pharmaceutical companies. An overview of currently active studies at the Van Creveldkliniek is shown in the table below.
| Study | Patient population | Description |
|---|---|---|
| ActivateKids | Pediatrics with non-transfusion dependent pyruvate kinase deficiency (PKD) | AG-348 (Mitapivat) in pediatric PKD patients. Start Q1 2023. |
| ActivateKidsT | Pediatrics with transfusion dependent pyruvate kinase deficiency (PKD) | AG-348 (Mitapivat) in pediatric PKD patients. Start Q4 2022. |
| PEAK | Pediatric and adult patients with PKD | Registry on PKD |
| PKD Natural History | Pyruvate kinase deficiency | Registry on PKD |
| ENERGIZE | Adults with non-transfusion dependent thalassemia | Evaluation of safety and efficacy of AG-348 (Mitapivat) |
| ENERGIZE-T | Adults with transfusion dependent thalassemia | Evaluation of safety and efficacy of AG-348 (Mitapivat) |
| ESTIMATE | Adults with sickle cell disease (SCD) | AG-348 (Mitapivat) in SCD |
| RISE-UP | Adults with sickle cell disease (SCD) | AG-348 (Mitapivat) in SCD |
| IronEry | Rare anemias | Investigating the interplay between (iron) overload and erythropoiesis in rare hereditary anemias. |
| SATISFY | Adults with erythrocyte membranopathies | Mitapivat (pyrukynd) in spherocytosis, xerocytosis and congenital dyserytropoetic anemia type 2 (CDA II) |
Contact
Do you have any questions about participation in one of our studies? Or do you want to get in touch with one of our researchers? Please feel free to contact the research team of the Van Creveldkliniek.