Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

Summary

This is a prospective exploratory multicentre pilot study designed to study the safety and efficacy of mitapivat in RBC membranopathies and CDAII

Timeline

Start

2023-12-21

Primary completion

2025-07-15

Completion

2026-12-15

Publications

• Glenthoj A, van Beers EJ, van Wijk R, Rab MAE, Groot E, Vejlstrup N, Toft N, Bendtsen SK, Petersen J, Helby J, Chermat F, Fenaux P, Kuo KHM. 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. BMJ Open. 2024 Jul 30;14(7):e083691. doi: 10.1136/bmjopen-2023-083691.

Drugs

Indications