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Pyruvate kinase deficiency anaemia

  • Study on the Effectiveness and Safety of Mitapivat for Children with Pyruvate Kinase Deficiency Receiving Regular Blood Transfusions

    Not recruiting

    3 1
    Investigated Drugs:
    • Mitapivat
    Czechia Denmark The Netherlands Spain

  • Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

    Not recruiting

    1 1 1 1
    Investigated Diseases:
    • Pyruvate kinase deficiency anaemia
    Investigated Drugs:
    • Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
    • Plerixafor
    • Filgrastim
    Spain

  • Study on the Effectiveness and Safety of Mitapivat in Children with Pyruvate Kinase Deficiency Not Receiving Regular Blood Transfusions

    Not recruiting

    3 1
    Investigated Diseases:
    • Pyruvate kinase deficiency anaemia
    Investigated Drugs:
    • Mitapivat
    France Germany Italy The Netherlands Spain

  • Study on Long-Term Safety of Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Pyruvate kinase deficiency anaemia
    Investigated Drugs:
    • Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
    Spain

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European Clinical Trials Information Network

The European Clinical Trials Information Network is transforming access to clinical trial information for patients across Europe. By overcoming language barriers, our platform allows everyone, regardless of their native language, to easily find relevant and comprehensible clinical trial data. This innovative service connects patients to cutting-edge treatments, creating a unified European community where health and progress transcend borders.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic centres, national regulators, and commercial sponsors. We aim to keep all content accurate and up to date.

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