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Thalassaemia

  • A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions

    Recruiting

    2 1 1 1
    Investigated Diseases:
    • Thalassaemia beta
    • Thalassaemia
    Investigated Drugs:
    • Lenograstim
    • Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
    • Plerixafor
    Italy

  • Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Thalassaemia
    Investigated Drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Busulfan
    Germany Italy

  • Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

    Recruiting

    3 1 1
    Investigated Diseases:
    • Thalassaemia
    • Sickle cell disease
    Investigated Drugs:
    • Etavopivat
    France Germany Greece Italy Spain

  • Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Thalassaemia
    • Primary myelofibrosis
    • Myelodysplastic syndrome
    Investigated Drugs:
    • Luspatercept
    Bulgaria France Germany Greece Italy The Netherlands +2

  • Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Sickle cell anaemia
    • Thalassaemia
    Investigated Drugs:
    • Exagamglogene Autotemcel
    Belgium Germany Italy

  • Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Thalassaemia beta
    • Thalassaemia
    Investigated Drugs:
    • Luspatercept
    The Netherlands
1 2
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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.

Explore the full ecosystem of clinical research in Europe.

The information provided on this website is mainly derived from international public clinical trial databases (CTIS.eu). In addition, we are including supplementary sources, such as information provided by academic centers and national regulators, as well as information supplied by commercial sponsors (pharmaceutical companies and contract research organizations). We try to ensure that the information provided is accurate and up to date.

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