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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

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

    Not yet recruiting

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

  • Comparing morphine alone to morphine and lidocaine for pain relief in sickle cell anemia patients with vaso-occlusive crisis

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    • Sickle cell anaemia
    • Sickle cell anaemia with crisis
    • Sickle cell disease
    Investigated drugs:
    • Lidocaine Hydrochloride
    • Morphine Hydrochloride
    Belgium

  • A Study Comparing Gadopiclenol and Gadoterate Meglumine for Heart MRI Scans in Children with Congenital Heart Disease

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    • Cardiomyopathy
    Investigated drugs:
    • Meglumine Gadoterate
    Spain

  • Study of sodium butyrate enemas for bowel function recovery after surgery in children and adults with Hirschsprung’s disease

    Not yet recruiting

    3 1 1 1
    Investigated drugs:
    • Sodium Butyrate
    France

  • A Study Testing the Safety and Tolerability of ALE1 in Healthy Adults and Adults with Hypophosphatasia

    Not yet recruiting

    2 1
    Investigated diseases:
    • Hypophosphatasia
    Investigated drugs:
    • ALE1
    Germany

  • A study of mirdametinib for infants and toddlers under 24 months of age with neurofibromatosis type 1 associated plexiform neurofibromas

    Not yet recruiting

    1 1 1
    Germany 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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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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