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New hope for rare blood disorder: Long-Term drug study shows promise

NCT ID NCT03853798

Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety and effectiveness of the drug mitapivat in 90 adults with pyruvate kinase deficiency, a rare genetic blood disorder that causes red blood cells to break down too quickly. Participants had already completed earlier studies of mitapivat and continued taking the drug to see how well it worked over time and what side effects occurred. The goal was to understand if mitapivat can safely help manage this lifelong condition.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

90 people

The number who actually took part.

Started

Mar 2019

Finished

Jul 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

pyruvate kinase deficiency of red cells

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pyruvate kinase deficiency

Contacts and locations

Locations

  • AORN Cardarelli

    Naples, 00165, Italy

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHU Hopitaux de Bordeaux - Hôpital Saint-André

    Bordeaux, 33000, France

  • CHU Hôpital Henri Mondor

    Créteil, 94010, France

  • Centre Hospitalier Universitaire Vaudois (CHUV)

    Vaud (Lausanne), 1011, Switzerland

  • Charite - UB - CVK - Medizinische Klinik

    Berlin, 10117, Germany

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Emory-Children's Center

    Atlanta, Georgia, 30322, United States

  • Faculty of Medicine Siriraj Hospital

    Bangkok, 10700, Thailand

  • Hacettepe University Faculty of Medicine

    Ankara, 06100, Turkey (Türkiye)

  • Herlev University Hospital

    Herlev, 2730, Denmark

  • Hospital La Timone

    Marseille, 13385, France

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital. U. Vall d'Hebron

    Barcelona, 08035, Spain

  • Houston Methodist Research Institute

    Houston, Texas, 77030, United States

  • Htal Clínico Universitario Virgen de la Arrixaca.

    Murcia, 30120, Spain

  • Imperial College Healthcare NHS Trust

    London, W2 INY, United Kingdom

  • Indiana Hemophilia & Thrombosis Center Inc.

    Indianapolis, Indiana, 46260, United States

  • Institut Universitaire du Cancer de Toulouse - Oncopole

    Toulouse, 31100, France

  • Kansai Medical University, Dep. of Pediatrics, Hirakata Hospital

    Osaka, 573-1010, Japan

  • Kyoto Katsura Hospital

    Kyoto, 615-8256, Japan

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • McMaster University

    Hamilton, Ontario, L8N3Z5, Canada

  • Mie University Hospital

    Tsu, Mie-ken, 514-8507, Japan

  • Osp Maggiore Policlinico Milano

    Milan, 20122, Italy

  • Ospedale Galliera

    Genova, 16128, Italy

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Seattle Cancer Care Alliance

    Seattle, Washington, 98195, United States

  • St James's Hospital

    Dublin, Ireland

  • Toho University Omori Medical Center

    Tokyo, 8541, Japan

  • Tohoku University Hospital

    Sendai, Miyagi, 980-8574, Japan

  • UCSF Benioff Children's Hospital, Oakland

    Oakland, California, 95609, United States

  • UNICAMP - Hemocentro

    São Paulo, 13083-878, Brazil

  • University College London

    London, WC1E 6BT, United Kingdom

  • University of Utah

    Salt Lake City, Utah, 84113, United States

  • Università della Campania "Luigi Vanvitelli"

    Naples, 80318, Italy

  • Universitätsklinik Würzburg

    Würzburg, 97080, Germany

  • Van Creveldkliniek

    Utrecht, 3508 GA, Netherlands

  • Wayne State University School of Medicine

    Detroit, Michigan, 48201, United States

  • Yeungnam University Hospital

    Daegu, 705-703, South Korea

More trials for these conditions

Other studies related to the condition(s) this trial covers.