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Research line 'Rare anemias and bone marrow failure'
Research line 'Rare anemias and bone marrow failure'
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. |
| AG348-C-011 | Adult pyruvate kinase deficiency patients (PKD) | AG-348 (Mitapivat) in adult PKD patients. |
| PEAK | Pediatric and adult patients with PKD | Registry on PKD. |
| PEAK-PRO | Adult PKD patients | Characterization of health-related quality of life and disease burden of PKD |
| PEAK COG | Adult PKD patients | Investigation of cognition in patients with 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. |
| IronEry | Rare anemias | Investigating the interplay between (iron) overload and erythropoiesis in rare hereditary anemias. |
| PPI Shine Again | Non-transfusion dependent hereditary anemias with iron overload | Efficacy of proton pump inhibitors in the prevention of iron overload. |
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.
Telefoonnummer: +31 (0)88 75 584 50
Email adres: vck-research@umcutrecht.nl