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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety and Effects of Luspatercept for Children with Beta Thalassemia

    Recruiting

    2 1 1 1
    Investigated Diseases:
    • Thalassaemia beta
    Investigated Drugs:
    • Luspatercept
    Germany Greece Italy

  • Study on the Use of Simoctocog Alfa and Emicizumab for Surgery in Patients with Severe Hemophilia A

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Simoctocog Alfa
    Croatia Finland France Germany Italy Spain

  • Study on the Use of Simoctocog Alfa for Women and Girls with Hemophilia A Undergoing Surgery

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor VIII deficiency
    Investigated Drugs:
    • Simoctocog Alfa
    Finland France Germany Italy Romania Spain

  • Study on Early Use of Candesartan vs Placebo for People with Genetic Risk of Dilated Cardiomyopathy

    Recruiting

    3 1 1
    Investigated Diseases:
    • Dilated cardiomyopathy
    Investigated Drugs:
    • Candesartan Cilexetil
    The Netherlands Spain

  • Study on the Effectiveness of Atenativ for Patients with Congenital Antithrombin Deficiency Undergoing Surgery or Childbirth

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Antithrombin Iii Human
    • Human Plasma Derived Antithrombin
    Austria Czechia France Germany Hungary Italy +2

  • Study on the Safety and Efficacy of Avalglucosidase Alfa for Patients Aged 5 and Older with Non-Classic Pompe Disease

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Glycogen storage disease type II
    Investigated Drugs:
    • Avalglucosidase Alfa
    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.

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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