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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety and Effects of Tolvaptan for Infants and Young Children with Autosomal Recessive Polycystic Kidney Disease

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Congenital cystic kidney disease
    Investigated Drugs:
    • Tolvaptan
    Belgium Germany Poland Spain

  • Long-term safety study of garadacimab (CSL312) for prevention of hereditary angioedema attacks

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Hereditary angioedema
    Investigated Drugs:
    • Garadacimab
    Czechia Germany Hungary The Netherlands Spain

  • Study on Long-Term Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Fragile X syndrome
    Investigated Drugs:
    • Cannabidiol
    Ireland

  • Study to Determine the Best Dose of Vesleteplirsen for Patients with Duchenne Muscular Dystrophy Suitable for Exon 51-Skipping Treatment

    Not recruiting

    2 1 1
    Investigated Drugs:
    • Vesleteplirsen
    Belgium Germany Italy The Netherlands Spain

  • Study on the Safety and Effects of PTC518 for Patients with Huntington’s Disease

    Not recruiting

    2 1
    Investigated Drugs:
    • Ptc518
    Austria France Germany Italy The Netherlands Spain

  • Study on NTLA-2002 for Adults with Hereditary Angioedema Using Ziclumeran and Lonvoguran

    Not recruiting

    1 1
    Investigated Drugs:
    • Lonvoguran
    • Ziclumeran
    France Germany The Netherlands
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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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