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Study identifier: NCT07055243 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Anemia  ·  Sponsor: University Health Network, Toronto

PhasePhase 2
Planned participants9
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Kevin Kuo, MD  ·  416-340-5233  ·  Kevin.Kuo@uhn.ca

Always discuss trial participation with your own doctor first.

Locations (1)

University Health Network (UHN)Toronto, Ontario, CanadaRecruiting

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Source record: clinicaltrials.gov/study/NCT07055243