Enrolling by invitation NCT05777993
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Mitapivat.
- Who it may be relevant to
- Registry conditions: Pyruvate Kinase Deficiency, Anemia, Hemolytic. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Canada, Japan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Overview
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.
Detailed description
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.
Interventions
- Drug Mitapivat
Tablets
Primary outcome measures
- Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time frame: Up to approximately 6.2 years]
- Percentage of Participants With AEs and SAEs, Graded by Severity [Time frame: Up to approximately 6.2 years]
- Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug [Time frame: Up to approximately 6.2 years]
- Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug [Time frame: Up to approximately 6.2 years]
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).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Japan · 3 centers
- Tohoku University Hospital — Sendai
- Kyoto Katsura Hospital — Kyoto
- Toho University - Omori Medical Center — Ōta-ku
Canada · 1 center
- McMaster Centre for Transfusion Medicine McMaster University — Hamilton
Identifiers
NCT: NCT05777993 · AG348-C-025