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Lifeline drug mitapivat keeps flowing for rare blood disorder patients

NCT ID NCT05777993

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 13, 2026 · Updated 2 times

Summary

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does not test new treatments but ensures those who benefited can keep taking mitapivat.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mitapivat (tablet)
What this could lead to
If successful, this study ensures uninterrupted treatment for people with pyruvate kinase deficiency who lack commercial access.
What could go wrong
This is a small rollover study (6 participants) focused on safety and continued access, not on proving new benefits. Side effects remain possible.

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 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 59 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2023

Expected to finish

Aug 2029

An estimate. End dates often move.

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: * Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator. * For women of childbearing potential (WOCBP), 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/assent, 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/assent from the participant (or their legally authorized representative, parent\[s\], or legal guardian) before any study-related procedures are conducted, and participants must be 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: * Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB). * 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 and/or could confound the interpretation of the study data. 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).

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Conditions

The condition(s) this trial relates to.

hemolytic anemia pyruvate kinase deficiency of red cells sickle cell disease thalassemia

As listed by the trial registrant

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

Anemia, hemolytic Pyruvate kinase deficiency Sickle cell disease Thalassemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    23 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AP-HP - Hôpital Européen Georges Pompidou

    Paris, 75015, France

  • Acibadem Adana Hospital

    Seyhan, Adana, 01130, Turkey (Türkiye)

  • CHU Hôpital Henri Mondor

    Créteil, 94010, France

  • CHU de Bordeaux - Hôpital Pellegrin

    Bordeau, 33000, France

  • GSH Banco de Sangue de São Paulo

    São Paulo, 04006-002, Brazil

  • HEMORIO Instituto Nacional de Hematologia

    Rio de Janeiro, 20211-030, Brazil

  • Hopital Edouard Herriot, CHU de Lyon

    Lyon, 69003, France

  • Hopitaux de La Timone

    Marseille, Bouches-du-Rhône, 13005, France

  • Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP

    São Paulo, 14051-030, Brazil

  • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

    Porto Alegre, Rio Grande do Sul, 90619-900, Brazil

  • Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo

    São Paulo, 05403-010, Brazil

  • Hospital de Clínicas da Unicamp

    Campinas, São Paulo, 13083-878, Brazil

  • Hôpital Erasme

    Anderlecht, Brussels Capital, 1070, Belgium

  • Hôpital Necker Enfants Malades

    Paris, 75015, France

  • Kyoto Katsura Hospital

    Kyoto, 615-8256, Japan

  • Manchester Royal Infirmary, Manchester University NHS Foundation Trust

    Manchester, M13 9W, United Kingdom

  • McMaster Centre for Transfusion Medicine McMaster University

    Hamilton, Ontario, L8S3Z5, Canada

  • Praxis Pesquisa Medica

    Santo André, 09090-790, Brazil

  • Rambam Medical Center

    Haifa, Ḥeifā, 31096, Israel

  • Toho University - Omori Medical Center

    Ōta-ku, 143-8541, Japan

  • Tohoku University Hospital

    Sendai, Miyagi, 980-8574, Japan

  • University Health Network

    Toronto, Ontario, M5G2C4, Canada

  • Ziv Medical Center

    Safed, Ḥeifā, 13100, Israel

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