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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Long-Term Safety Study of Everolimus for Patients with Tuberous Sclerosis Complex and Refractory Seizures

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Tuberous sclerosis complex
    Investigated Drugs:
    • Everolimus
    Poland
  • Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor VIII deficiency
    Investigated Drugs:
    • Efanesoctocog Alfa
    Italy Norway Spain Sweden
  • Study on the Effects of Sodium Valproate for Hearing Preservation in Patients with Wolfram Syndrome

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Wolfram syndrome
    Investigated Drugs:
    • Valproic Acid
    • Sodium Valproate
    France Spain
  • Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

    Not recruiting

    2 1 1
    Investigated Drugs:
    • Anakinra
    Germany
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Haemophilia A with anti factor VIII
    • Haemophilia B with anti factor IX
    Investigated Drugs:
    • Concizumab
    Croatia Denmark France Italy Poland Portugal +2
  • Long-term Study on the Safety and Effectiveness of Odevixibat for Patients with Alagille Syndrome

    Not recruiting

    3 1 1 1
    Investigated Drugs:
    • Odevixibat
    Belgium France Germany Italy The Netherlands Poland
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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.

Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).

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