Hereditary Red Blood Cell Disorder (Disorder) Clinical Trial
Official title:
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
This is a prospective exploratory multicentre pilot study designed to study the safety and efficacy of mitapivat in RBC membranopathies and CDAII
Patients will be recruited from centres of expertise in the European Union starting with Denmark and the Netherlands, and outside the EU in Canada. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada. For this purpose, two sibling studies will be conducted: One in the EU and one in Canada. The study report will be a combined analysis of all patients (EU + Canada) where some exploratory measures will only be available for EU patients. This protocol is for the EU portionof the study This project is carried out within the framework of European Reference Network on Rare Haematological Diseases (ERN-EuroBloodNet)-Project ID No 101085717. ERN-EuroBloodNet is partly co-funded by the European Union within the framework of the Fourth EU Health Programme. ;