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

Agios Pharmaceuticals

Status and phase

Invitation-only

Phase 4

Conditions

Thalassemia

Anemia, Hemolytic

Sickle Cell Disease

Pyruvate Kinase Deficiency

Treatments

Drug: Mitapivat

Study type

Interventional

Funder types

Industry

Identifiers

NCT05777993

AG348-C-025

2026-526243-35-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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.

Full description

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

Enrollment

59 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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).