A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

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

The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.

Study Overview

• Status: Enrolling by invitation
• Conditions: Sickle Cell Disease; Anemia, Hemolytic; Thalassemia; 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): 59
• Phase: Phase 4

Contacts and Locations

Study Locations

• Belgium
  • Brussels Capital
    • Anderlecht, Brussels Capital, Belgium, 1070
      • Hopital Erasme
• Brazil
  • Rio de Janeiro, Brazil, 20211-030
    • HEMORIO Instituto Nacional de Hematologia
  • Santo André, Brazil, 09090-790
    • Praxis Pesquisa Medica
  • São Paulo, Brazil, 04006-002
    • GSH Banco de Sangue de São Paulo
  • São Paulo, Brazil, 05403-010
    • Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
  • São Paulo, Brazil, 14051-030
    • Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
  • Rio Grande do Sul
    • Porto Alegre, Rio Grande do Sul, Brazil, 90619-900
      • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
  • São Paulo
    • Campinas, São Paulo, Brazil, 13083-878
      • Hospital de Clinicas da UNICAMP
• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8S3Z5
      • McMaster Centre for Transfusion Medicine McMaster University
    • Toronto, Ontario, Canada, M5G2C4
      • University Health Network
• France
  • Bordeau, France, 33000
    • CHU de Bordeaux - Hôpital Pellegrin
  • Créteil, France, 94010
    • CHU Hôpital Henri Mondor
  • Lyon, France, 69003
    • Hopital Edouard Herriot, CHU de Lyon
  • Paris, France, 75015
    • Hôpital Necker Enfants Malades
  • Paris, France, 75015
    • AP-HP - Hôpital Européen Georges Pompidou
  • Bouches-du-Rhône
    • Marseille, Bouches-du-Rhône, France, 13005
      • Hopitaux de La Timone
• Israel
  • Ḥeifā
    • Haifa, Ḥeifā, Israel, 31096
      • Rambam Medical Center
    • Safed, Ḥeifā, Israel, 13100
      • ZIV Medical Center
• Japan
  • Kyoto, Japan, 615-8256
    • Kyoto Katsura Hospital
  • Ōta-ku, Japan, 143-8541
    • Toho University - Omori Medical Center
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital
• Turkey (Türkiye)
  • Adana
    • Seyhan, Adana, Turkey (Türkiye), 01130
      • Acibadem Adana Hospital
• United Kingdom
  • Manchester, United Kingdom, M13 9W
    • Manchester Royal Infirmary, Manchester University NHS Foundation Trust

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator.
• For women of childbearing potential (WOCBP), 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/assent, 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/assent from the participant (or their legally authorized representative, parent[s], or legal guardian) before any study-related procedures are conducted, and participants must be 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:

• Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB).

• 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 and/or could confound the interpretation of the study data. 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 continue to receive mitapivat at the same dose regimen they were receiving at the final visit of their antecedent study.	Drug: Mitapivat; Tablets, granules; Other Names: AG-348; Mitapivat sulfate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study	Not Applicable - Study Completion

Secondary 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 6, 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): August 12, 2026
• Last Update Submitted That Met QC Criteria: August 10, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; Thalassemia; Anemia, Hemolytic; Pyruvate Kinase Deficiency of Red Cells; Molecular Mechanisms of Pharmacological Action; Enzyme Activators; mitapivat
• Other Study ID Numbers: AG348-C-025; 2026-526243-35-00 (Ctis)

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