Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT07055243

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

Led by University Health Network, Toronto · Updated on 2025-07-08

9

Participants Needed

1

Research Sites

104 weeks

Total Duration

On this page

Sponsors

U

University Health Network, Toronto

Lead Sponsor

A

Agios Pharmaceuticals, Inc.

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female diagnosed with RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII) confirmed by genetic testing
  • Age 18 years or older
  • Average hemoglobin below 13.0 g/dL for males and below 11.0 g/dL for females, with additional symptoms if hemoglobin is above 10.0 g/dL
  • Must start or continue taking at least 0.8 mg oral folic acid daily during the study
  • Adequate organ function including specific liver enzyme and kidney function levels
  • Willing and able to provide written informed consent and comply with study procedures
  • Women of reproductive potential must have a negative pregnancy test during screening
  • Women of reproductive potential must be abstinent or use highly effective contraception plus an additional barrier method during and for 28 days after the study
Not Eligible

You will not qualify if you...

  • History of pyruvate kinase deficiency
  • Receiving regular blood transfusions exceeding 5 in the past year or within 3 months before treatment
  • Significant medical conditions such as uncontrolled hypertension, heart disease, symptomatic gallbladder issues, drug-induced hepatitis, severe iron overload, other blood disorders, or active infections
  • Positive tests for active hepatitis B or C or HIV
  • Poorly controlled diabetes requiring multiple medications
  • History of certain cancers unless treated and disease-free for 3 years
  • Unstable extramedullary hematopoiesis or severe liver disease
  • Recent major surgery within 6 months
  • Current participation in another therapeutic clinical trial
  • Exposure to investigational drugs within 3 months before treatment
  • Prior treatment with pyruvate kinase activators
  • Previous bone marrow or stem cell transplant
  • Pregnancy, breastfeeding, or planning pregnancy during the study
  • Use of strong CYP3A4 inhibitors or inducers without appropriate washout
  • Use of hematopoietic stimulating agents without 28-day washout
  • Known allergy to mitapivat or its components
  • Unwillingness to use required contraception or abstinence
  • Use of unstable herbal or dietary supplements within 8 weeks prior to randomization

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

University Health Network (UHN)

Toronto, Ontario, Canada, M5G 2C4

Actively Recruiting

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Research Team

K

Kevin Kuo, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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