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

NCT07055243 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2025-07-08

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Mitapivat

Mitapivat will be given to adult patients with erythrocyte membranopathies

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • University Health Network, Toronto

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-26
Primary Completion
2026-06-30
Completion
2027-06-30

Countries

  • Canada

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07055243 on ClinicalTrials.gov