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A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Sponsor
Agios Pharmaceuticals, Inc. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05777993
Collaborator
(none)
10
Enrollment
4
Locations
1
Arm
74
Anticipated Duration (Months)
2.5
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
Phase 4

Detailed Description

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

Study Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Anticipated Study Start Date :
Jun 1, 2023
Anticipated Primary Completion Date :
Aug 1, 2029
Anticipated Study Completion Date :
Aug 1, 2029

Arms and Interventions

Arm Intervention/Treatment
Experimental: Mitapivat

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
Tablets
Other Names:
  • AG-348
  • Mitapivat sulfate
  • PYRUKYND®

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Up to approximately 6.2 years]

    2. 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.

    3. Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug [Up to approximately 6.2 years]

    4. Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug [Up to approximately 6.2 years]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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).

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 McMaster University Hamilton Ontario Canada L8S 4K1
    2 Kyoto Katsura Hospital Kyoto-Shi Japan 615-8256
    3 Toho University - Omori Medical Center Ota-Ku Japan 143-8541
    4 Tohoku University Hospital Sendai-Shi Japan 980-8574

    Sponsors and Collaborators

    • Agios Pharmaceuticals, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Agios Pharmaceuticals, Inc.
    ClinicalTrials.gov Identifier:
    NCT05777993
    Other Study ID Numbers:
    • AG348-C-025
    First Posted:
    Mar 21, 2023
    Last Update Posted:
    Mar 21, 2023
    Last Verified:
    Mar 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Anemia, Hemolytic

    Study Results

    No Results Posted as of Mar 21, 2023