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

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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Anemia, Hemolytic; Pyruvate Kinase Deficiency
• Intervention / Treatment: Drug: Mitapivat

Detailed Description

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

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Agios Medical Affairs; Phone Number: 833-228-8474; Email: medinfo@agios.com

Study Locations

• Japan
  • Kyoto, Japan
    • Kyoto Katsura Hospital
  • Ota-Ku, Japan, 143-8541
    • Toho University - Omori Medical Center
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mitapivat; 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; Tablets; Other Names: AG-348; Mitapivat sulfate; PYRUKYND®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Agios Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 5, 2023
• Primary Completion (Estimated): August 1, 2029
• Study Completion (Estimated): August 1, 2029

Study Registration Dates

• First Submitted: March 9, 2023
• First Submitted That Met QC Criteria: March 9, 2023
• First Posted (Actual): March 21, 2023

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Anemia; Anemia, Hemolytic; Molecular Mechanisms of Pharmacological Action; Enzyme Activators; Mitapivat
• Other Study ID Numbers: AG348-C-025

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No