Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol

Andreas Glenthøj(University of Copenhagen), Kevin H.M. Kuo(University Health Network), Fatiha Chermat(Groupe Francophone des Myélodysplasies), Niels Vejlstrup(Copenhagen University Hospital), Minke A.E. Rab(Utrecht University), Jesper Petersen(Copenhagen University Hospital), Richard van Wijk(Utrecht University), Jens Helby(University of Copenhagen), Selma Kofoed Bendtsen(Copenhagen University Hospital), Nina Toft(Copenhagen University Hospital), Eduard J. van Beers(Utrecht University), Evelyn Groot(University Medical Center Utrecht), Pierre Fenaux(Saint Louis University Hospital)
BMJ Open
July 1, 2024
Cited by 11


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