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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study comparing venglustat tablets with standard therapy for adult patients with Fabry disease and left ventricular hypertrophy

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Fabry’s disease
    Investigated Drugs:
    • AGALSIDASE BETA
    • Migalastat
    • Venglustat
    Austria Czechia Denmark France Germany Greece +5

  • Study of alpelisib in children and adults with PIK3CA-Related Overgrowth Spectrum (PROS)

    Not recruiting

    2 1
    Investigated Drugs:
    • Alpelisib
    France Germany Italy The Netherlands Norway Spain

  • Safety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema

    Not recruiting

    3 1 1
    Investigated Drugs:
    • Garadacimab
    Germany

  • Study of ISIS 678354 for Patients with Severe High Triglycerides

    Not recruiting

    3 1
    Investigated Diseases:
    • Familial hypertriglyceridaemia
    • Hyperchylomicronaemia
    Investigated Drugs:
    • Isis 678354 Sodium Salt
    Belgium Bulgaria Czechia France Greece Hungary +9

  • Study on the Effectiveness of Nintedanib for Treating Nosebleeds in Patients with Hereditary Hemorrhagic Telangiectasia

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Hereditary haemorrhagic telangiectasia
    Investigated Drugs:
    • Nintedanib
    France

  • Study on the Safety and Effectiveness of Zagociguat for Treating MELAS in Patients

    Not recruiting

    2 1
    Investigated Drugs:
    • Zagociguat
    Germany Italy
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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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