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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on VX-522 and Ivacaftor for Adults with Cystic Fibrosis Unresponsive to CFTR Modulator Therapy

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Cystic fibrosis
    Investigated Drugs:
    • Vx-522
    • Ivacaftor
    Belgium Germany Italy The Netherlands Spain Sweden

  • Study on the Effects of Romosozumab and Bisphosphonates in Children and Adolescents with Osteogenesis Imperfecta

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Osteogenesis imperfecta
    Investigated Drugs:
    • Calcium
    • Ergocalciferol
    • Romosozumab
    Austria Belgium France Germany Hungary Italy +3

  • Study on the Safety and Effects of STAR-0215 for Adults with Hereditary Angioedema

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Hereditary angioedema with C1 esterase inhibitor deficiency
    • Hereditary angioedema
    Investigated Drugs:
    • Star-0215
    Bulgaria Czechia Germany Poland

  • Study on the Safety and Effects of AMB-05X Injections for Patients with Tenosynovial Giant Cell Tumor

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Synovitis
    Investigated Drugs:
    • Amb-05X
    The Netherlands

  • Study on the Safety and Effects of Garadacimab for Preventing Hereditary Angioedema in Children Aged 2 to 11

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Hereditary angioedema
    Investigated Drugs:
    • Garadacimab
    Germany Italy

  • Study on the Safety and Effects of Setanaxib for Patients with Alport Syndrome

    Not recruiting

    2 1
    Investigated Diseases:
    • Alport’s syndrome
    Investigated Drugs:
    • Setanaxib
    Austria Czechia France Lithuania Slovakia Spain
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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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