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

Pyruvate Kinase Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05777993
• Other study ID #: AG348-C-025
• Status: Enrolling by invitation
• Phase: Phase 4
• Start date: July 5, 2023
• Est. completion date: August 2029

Study information

• Verified date: May 2024
• Source: Agios Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 6
• Est. completion date: August 2029
• Est. primary completion date: August 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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).

Related Conditions & MeSH terms

• Anemia, Hemolytic
• Pyruvate Kinase Deficiency

Intervention

Drug:
• Mitapivat
Tablets

Locations

Country	Name	City	State
Japan	Kyoto Katsura Hospital	Kyoto
Japan	Toho University - Omori Medical Center	Ota-Ku
Japan	Tohoku University Hospital	Sendai	Miyagi

Sponsors (1)

Lead Sponsor	Collaborator
Agios Pharmaceuticals, Inc.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		Up to approximately 6.2 years
Primary	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
Primary	Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug		Up to approximately 6.2 years
Primary	Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug		Up to approximately 6.2 years