A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

NCT05777993 | Enrolling By Invitation Phase 4 FDA Drug

• 1 other identifier: AG348-C-025
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 4

Brief Summary

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.

Conditions

Pyruvate Kinase Deficiency; Anemia, Hemolytic

Outcome Measures

Primary Outcomes (4)

• Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Up to approximately 6.2 years

• Percentage of Participants With AEs and SAEs, Graded by Severity

  The severity of all AEs will be graded by the Investigator according to Version 4.03 of the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) on a 5-point severity scale (Grade 1 through Grade 5) where Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening and Grade 5 is fatal.

  Up to approximately 6.2 years

• Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug

  Up to approximately 6.2 years

• Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug

  Up to approximately 6.2 years

Study Arms (1)

Mitapivat

EXPERIMENTAL

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.

Drug: Mitapivat

Interventions

Mitapivat DRUG

Tablets

Also known as: AG-348, Mitapivat sulfate, PYRUKYND®

Mitapivat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has completed an Agios-sponsored adult pyruvate kinase (PK) deficiency study of mitapivat within 1 day of enrollment in this rollover study;
• Is unable to obtain mitapivat treatment due to country regulations or lack of commercial access;
• Was deriving clinical benefit from mitapivat treatment at the completion of the antecedent study, in the opinion of the Investigator;
• 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;
• 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;
• Agrees not to participate in other interventional clinical studies, including other studies of mitapivat, during participation in this study.

You may not qualify if:

• Has discontinued treatment with mitapivat before completing the antecedent study;
• Known allergy or other contraindication to mitapivat or its inactive ingredients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, Opadry II Blue \[hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD\&C Blue #2\]);
• Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study also excluded are:
• Participants who are institutionalized by regulatory or court order
• 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).

Sponsors & Collaborators

• Agios Pharmaceuticals, Inc.: lead

Study Sites (4)

McMaster Centre for Transfusion Medicine McMaster University

Hamilton, Ontario, L8S3Z5, Canada

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

Kyoto Katsura Hospital

Kyoto, 615-8256, Japan

Location

Toho University - Omori Medical Center

Ōta-ku, 143-8541, Japan

Location

MeSH Terms

Conditions

Pyruvate Kinase Deficiency of Red Cells; Anemia, Hemolytic

Interventions

mitapivat

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2023
• First Posted: March 21, 2023
• Study Start: July 6, 2023
• Primary Completion (Estimated): August 1, 2029
• Study Completion (Estimated): August 1, 2029
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

JP (3); CA (1)