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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Effectiveness and Safety of Avalglucosidase Alfa for Babies with Infantile-Onset Pompe Disease

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Glycogen storage disease type II
    Investigated Drugs:
    • Avalglucosidase Alfa
    Belgium Germany Italy The Netherlands Spain

  • Study on Long-term Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Adults with Late-onset Pompe Disease

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Glycogen storage disease type II
    Investigated Drugs:
    • Cipaglucosidase Alfa
    • Miglustat
    Belgium Denmark France Greece Hungary Italy +3

  • Study on the Safety and Tolerability of ST-920 Gene Therapy for Patients with Fabry Disease

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Fabry’s disease
    Investigated Drugs:
    • Isaralgagene Civaparvovec
    Germany Italy

  • Study on Long-Term Safety and Effects of PTC518 for Patients with Huntington’s Disease

    Not recruiting

    2 1
    Investigated Diseases:
    • Huntington’s disease
    Investigated Drugs:
    • VOTOPLAM
    Austria France Germany Italy The Netherlands Spain

  • Study on the Safety and Effects of SAR442501 for Children with Achondroplasia

    Not recruiting

    2 1 1
    Czechia Italy Spain

  • Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Adrenogenital syndrome
    Investigated Drugs:
    • Crn04894
    • Fludrocortisone
    • Abiraterone Acetate
    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.

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

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