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

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Mitapivat sulfate — DRUG
Subjects enrolled Maximum dose: will receive Mitapivat during 56 weeks Starting dose: 50 mg BD Maximum dose: 100 mg BD

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

Experimental: Mitapivat
subjects who enter the Dose Escalation Period will receive an initial dose of 50 mg mitapivat BID. After 4 weeks dose will be increased from 50 mg BID to 100 mg BID unless dose-limiting side effects have occurred or maximum allowed Hb levels have been reached. Subjects who safely tolerate mitapivat may be eligible to continue in two consecutive 24-week Fixed Dose Periods, allowing patients to remain on their tolerated dose of mitapivat for up to 48 weeks after dose escalation. During the Fixed Dose Periods, the dose may not exceed the maximum dose that was used during Dose Escalation Period. 8 weeks: Dose Escalation Period 24-week: Fixed Dose Period 1 24-week: Fixed Dose Period 2

Safety and tolerability of mitapivat in adult patients with erythrocyte membranopathies [ Time Frame: Up to 56 weeks ]