Clinical Trials Logo

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

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

Study information

Verified date June 2024
Source Agios Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
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).

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Mitapivat
Tablets

Locations

Country Name City State
Canada McMaster Centre for Transfusion Medicine McMaster University Hamilton Ontario
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.

Countries where clinical trial is conducted

Canada,  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
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03853798 - Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Phase 3
Completed NCT03559699 - A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
Active, not recruiting NCT02476916 - A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency Phase 2
Active, not recruiting NCT04105166 - Gene Therapy for Pyruvate Kinase Deficiency (PKD) Phase 1
Terminated NCT04964323 - Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Completed NCT04995315 - Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
Completed NCT03866590 - Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Recruiting NCT04902833 - Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Completed NCT02053480 - Pyruvate Kinase Deficiency Natural History Study
Completed NCT03548220 - A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
Not yet recruiting NCT06422351 - Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency Phase 2
Recruiting NCT03481738 - Pyruvate Kinase Deficiency Global Longitudinal Registry