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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study to Evaluate the Effectiveness of Spesolimab in Treating Patients with Netherton Syndrome

    Not recruiting

    4 1
    Investigated Diseases:
    • Netherton’s syndrome
    Investigated Drugs:
    • Spesolimab
    Austria Belgium Bulgaria Czechia Finland France +5

  • Study on Darolutamide and Androgen Deprivation Therapy for Men with Metastatic Hormone-Sensitive Prostate Cancer

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Prostate cancer metastatic
    Investigated Drugs:
    • Darolutamide
    Latvia Lithuania Spain

  • Study on Fitusiran for Preventing Bleeding in Males Aged 12 and Older with Severe Hemophilia A or B, with or without Inhibitors

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Haemophilia
    Investigated Drugs:
    • Damoctocog Alfa Pegol
    • Factor Viii Inhibitor Bypassing Fraction
    • Fitusiran
    France Germany Greece Italy Poland Spain

  • Study on Long-Term Safety and Efficacy of Gene Therapy for Leukocyte Adhesion Deficiency-I Using RP-L201 in Patients with LAD-I

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Leukocyte adhesion deficiency
    Investigated Drugs:
    • Autologous Cd34+ Haematopoietic Stem Cells Genetically Modified With Lentiviral Vector Encoding The Cd18 Gene
    Spain

  • Study on the Safety of DS-2325a for Patients with Netherton Syndrome

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Netherton’s syndrome
    Investigated Drugs:
    • Ds-2325A
    • Sodium Chloride Solution 0.9%
    France

  • Study on the Safety and Tolerability of VGL101 for Adults with Adult-Onset Leukoencephalopathy

    Not recruiting

    4 1 1
    Investigated Diseases:
    • Leukoencephalopathy
    Investigated Drugs:
    • Vgl101
    France Germany The Netherlands
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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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