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

Sponsor
University Health Network, Toronto
Study ID
NCT07055243
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Mitapivat — DRUG
    Mitapivat will be given to adult patients with erythrocyte membranopathies

Study Details

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.

Key Dates

Start date
Jun 26, 2025
Status verified
Jul 2025
Primary completion
Jun 30, 2026
Completion
Jun 30, 2027

Study Design

Enrollment
9 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Mitapivat
    All patients will receive the drug Mitapivat

Primary Outcome Measure

To evaluate safety of mitapivat [ Time Frame: 61 weeks ]

Central Contacts

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