A Study to Provide Continued Access to Mitapivat for Part... | NCT05777993

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Mitapivat	Drug	Tablets	Mitapivat	AG-348 Mitapivat sulfate

Outcomes Module

Primary Outcomes

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

Eligibility Module

Eligibility Criteria

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).

Facility	Status	City	State	ZIP	Country	Contacts
McMaster Centre for Transfusion Medicine McMaster University		Hamilton	Ontario	L8S3Z5	Canada
Tohoku University Hospital

ID	Term
C564858	Pyruvate Kinase Deficiency of Red Cells
D000743	Anemia, Hemolytic

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases