Anemia, Hemolytic, Pyruvate Kinase Deficiency, Sickle Cell Disease, Thalassemia
Conditions
Brief summary
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.
Detailed description
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.
Interventions
DRUGMitapivat
Tablets, granules
Sponsors
Agios Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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).
Design outcomes
Primary
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | Up to approximately 6.2 years | 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. |
| 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 | — |
Countries
Belgium, Brazil, Canada, France, Israel, Japan, Turkey (Türkiye), United Kingdom
Outcome results
None listed