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Lifeline drug mitapivat keeps flowing for rare blood disorder patients

NCT ID NCT05777993

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

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.

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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

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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