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

Primary Purpose

Pyruvate Kinase Deficiency, Anemia, Hemolytic

Status
Enrolling by invitation
Phase
Phase 4
Locations
Japan
Study Type
Interventional
Intervention
Mitapivat
Sponsored by
Agios Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pyruvate Kinase Deficiency

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Tohoku University Hospital
  • Toho University - Omori Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Mitapivat

Arm Description

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.

Outcomes

Primary Outcome Measures

Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug

Secondary Outcome Measures

Full Information

First Posted
March 9, 2023
Last Updated
October 12, 2023
Sponsor
Agios Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05777993
Brief Title
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Official Title
A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
July 5, 2023 (Actual)
Primary Completion Date
August 2029 (Anticipated)
Study Completion Date
August 2029 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Agios Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
Detailed Description
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pyruvate Kinase Deficiency, Anemia, Hemolytic

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Mitapivat
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Mitapivat
Other Intervention Name(s)
AG-348, Mitapivat sulfate, PYRUKYND®
Intervention Description
Tablets
Primary Outcome Measure Information:
Title
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Up to approximately 6.2 years
Title
Percentage of Participants With AEs and SAEs, Graded by Severity
Description
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.
Time Frame
Up to approximately 6.2 years
Title
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
Time Frame
Up to approximately 6.2 years
Title
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug
Time Frame
Up to approximately 6.2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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 Information:
Facility Name
Tohoku University Hospital
City
Sendai
State/Province
Miyagi
ZIP/Postal Code
980-8574
Country
Japan
Facility Name
Toho University - Omori Medical Center
City
Ota-Ku
ZIP/Postal Code
143-8541
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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