NCT07055243

To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies - Canada (SATISFY-CA)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: University Health Network, Toronto · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Patients will be in the trial for 57 weeks treatment weeks and a safety follow up week after 30 days from last dose. First 8 weeks will be dose escalating period followed by 48 weeks of fixed dose period. 57th week will be dose tapering week. Data collected from Princes Margaret will be incorporated to the main study conducted in EU for analysis. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada.

Detailed description

This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII. Gobal target is 25 patients. 9 patients from UHN, Canada will be enrolled to the study. All enrolled patients will receive the study drug Mitapivat. All eligible 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. The maximum dosage is 100 mg BID, for patients in which no dosage limiting side effects have occurred and maximum allowed hemoglobin levels have not been reached. Maximal study duration per subject is screening period (≤50 days) + 57 weeks on study drug: * 8 weeks Dose Escalation Period * 24 Fixed Dose Period 1 * 24 Fixed Dose Period 2 * 1 week Dose Taper Dose reduction is allowed at any time during both the Dose Escalation Period and the Fixed Dose Extension Period. In cases, where 50 mg BID provides dose limiting side effect(s), subjects can be offered dose reduction to 20 mg BID. If 20 mg BID provides dose limiting side effect(s), subjects can be further dose reduced to 5 mg BID. Subjects receiving dosage of \<50mg BID are allowed to continue in the study. Physical exam, ECG, vital signs, Dexa scan, MRI scan, safety blood work will be carried out thorough out the study. Patients will be asked to complete 2 questionnaires in a online web browser during the study visits at the clinic.

Conditions

• Anemia

Interventions

Type	Name	Description
DRUG	Mitapivat	Mitapivat will be given to adult patients with erythrocyte membranopathies

Timeline

• Start date: 2025-06-26
• Primary completion: 2026-06-30
• Completion: 2027-06-30
• First posted: 2025-07-08
• Last updated: 2025-07-08

Locations

1 site across 1 country: Canada