Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Completed · Phase 3

Conditions studied: Pyruvate Kinase Deficiency

In brief

This is an open-label, multicenter, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.

Key facts

Study ID

NCT03853798

Run by

Agios Pharmaceuticals, Inc.

People needed

90

Starts

2019-03-21

Expected to finish

2024-07-03

Last updated by the study team

2025-11-18

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Be willing and able to comply with study visits and procedures.
• Have signed written informed consent prior to participating in this extension study.
• Have completed either antecedent study AG348-C-006 or AG348-C-007 through the Part 2 Week 24 Visit.
• Cohorts 2 and 3: Have demonstrated clinical benefit from mitapivat treatment in the antecedent study, in the opinion of the Investigator.
• For women of reproductive potential, have a negative pregnancy test during screening of this extension study.
• For women of reproductive potential as well as men with partners who are women of reproductive potential, be abstinent 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 giving informed consent, during the study, and for 28 days following the last dose of study drug for women and 90 days following the last dose of study drug for men.

You may not qualify if…

• Have a significant medical condition (including clinically significant laboratory abnormality) that developed during his/her antecedent AG- 348 study that confers an unacceptable risk to participating in this extension study, that could confound the interpretation of the study data, and/or that compromises the ability of the participant to complete study visits and procedures.
• Are currently pregnant or breastfeeding.
• Have a splenectomy scheduled during the study treatment period.
• Meet the withdrawal criteria of his/her antecedent mitapivat study during screening of this extension study.
• Are currently receiving medications that are strong inhibitors of cytochrome P450 (CYP)3A4 that have not been stopped for a duration of at least 5 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug; or strong inducers of CYP3A4 that have not been stopped for a duration of at least 28 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug on this extension study.
• Have received anabolic steroids, including testosterone preparations, within 28 days prior to start of study drug on this extension study.
• Have received hematopoietic stimulating agents (eg, erythropoietins, granulocyte colony stimulating factors, thrombopoietins) within 28 days prior to start of study drug on this extension study.
• Have exposure to any investigational drug other than mitapivat, device, or procedure within 3 months prior to start of study drug on this extension study.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• UCSF Benioff Children's Hospital, Oakland — Oakland, California, United States
• Emory-Children's Center — Atlanta, Georgia, United States
• Indiana Hemophilia & Thrombosis Center Inc. — Indianapolis, Indiana, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Wayne State University School of Medicine — Detroit, Michigan, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Houston Methodist Research Institute — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Seattle Cancer Care Alliance — Seattle, Washington, United States
• UNICAMP - Hemocentro — São Paulo, Brazil
• McMaster University — Hamilton, Ontario, Canada
• Herlev University Hospital — Herlev, Denmark
• CHU Hopitaux de Bordeaux - Hôpital Saint-André — Bordeaux, France
• CHU Hôpital Henri Mondor — Créteil, France
• Hospital La Timone — Marseille, France
• Institut Universitaire du Cancer de Toulouse - Oncopole — Toulouse, France
• Charite - UB - CVK - Medizinische Klinik — Berlin, Germany
• Universitätsklinik Würzburg — Würzburg, Germany
• St James's Hospital — Dublin, Ireland
• Ospedale Galliera — Genova, Italy
• Osp Maggiore Policlinico Milano — Milan, Italy
• AORN Cardarelli — Naples, Italy

Full record on ClinicalTrials.gov

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