Continued Access to Mitapivat for Pyruvate Kinase Deficiency

Recruiting at 6 trial locations

Overseen By Agios Medical Affairs

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Agios Pharmaceuticals, Inc.

Must be taking: Mitapivat

Disqualifiers: Allergy to mitapivat, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to end of treatment period

6 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to provide continued access to mitapivat for participants who completed an Agios-sponsored mitapivat study (antecedent) and do not have commercial access to mitapivat.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the study team or your doctor.

What data supports the effectiveness of the drug Mitapivat for pyruvate kinase deficiency?

Mitapivat has been shown to improve hemoglobin levels and reduce the need for blood transfusions in adults with pyruvate kinase deficiency, a condition that causes anemia. It also improves patients' quality of life and has a generally good safety profile. ^{1 2 3 4 5}

Is mitapivat safe for humans?

Mitapivat has been generally well tolerated in clinical trials for adults with pyruvate kinase deficiency, with most side effects being mild to moderate in severity. ^{1 2 5 6 7}

How is the drug Mitapivat unique for treating pyruvate kinase deficiency?

Mitapivat is unique because it is the first approved drug that directly targets the underlying cause of pyruvate kinase deficiency by activating the pyruvate kinase enzyme in red blood cells, which helps improve hemoglobin levels and reduce the need for blood transfusions. ^{1 2 3 5 7}

Eligibility Criteria

This trial is for individuals who have completed a previous Agios-sponsored study on mitapivat and benefited from it, but can't get the drug commercially due to regulations or access issues. They must agree to use two forms of contraception if applicable, not join other studies, and comply with all procedures.

Inclusion Criteria

Is unable to obtain mitapivat treatment due to country regulations or lack of commercial access

Written informed consent from the participant before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study

For women of childbearing potential, must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method

Exclusion Criteria

Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor)

I am not allergic to mitapivat or its components.

Participants who are institutionalized by regulatory or court order

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue the mitapivat dose regimen they were receiving at the final visit of their antecedent study

Up to end of treatment period

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to approximately 6.2 years

Treatment Details

Interventions

• Mitapivat

Trial Overview The trial provides continued access to mitapivat for participants with hemolytic anemia or pyruvate kinase deficiency who previously responded well in an earlier study but lack commercial access to the medication.

Participant Groups

Group I: Mitapivat Experimental Treatment 1 Intervention

Participants will receive mitapivat of 5, 20 or 50 milligrams (mg), orally, twice daily (BID), based on the last dose received by the participants in the antecedent study. Mitapivat will be administered from Day 1 up to end of treatment period of this study.

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+

References

1. England pubmed.ncbi.nlm.nih.gov

Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Mitapivat versus Placebo for Pyruvate Kinase Deficiency. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Pyruvate kinase activators for treatment of pyruvate kinase deficiency. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults. [2023]

6. Germany pubmed.ncbi.nlm.nih.gov

Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial. [2023]

7. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency. [2023]