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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on ECUR-506A and ECUR-506D for Male Infants Under 9 Months with Neonatal Onset Ornithine Transcarbamylase Deficiency

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Ornithine transcarbamoylase deficiency
    Investigated Drugs:
    • Adeno-Associated Virus Serotype Rh79 Containing The Human Otc Gene
    Spain

  • Study on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy

    Not recruiting

    2 1 1
    Denmark Italy Poland Spain

  • Study on the Effect of Allopurinol and Hypothermia for Newborns with Hypoxic-Ischemic Encephalopathy

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Encephalopathy neonatal
    • Neonatal asphyxia
    Investigated Drugs:
    • Allopurinol Sodium
    • Mannitol
    Austria Belgium Estonia Finland Germany Italy +3

  • Study of Fluconazole Treatment for Patients with Congenital Nephrogenic Diabetes Insipidus

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Congenital nephrogenic diabetes insipidus
    Investigated Drugs:
    • Clotrimazole
    Denmark

  • Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Usher’s syndrome
    Investigated Drugs:
    • Lentiviral Vector Derived From The Equine Infectious Anaemia Virus Containing The Human Myo7A Gene
    France

  • Study on the Long-Term Safety and Effectiveness of Pegunigalsidase Alfa for Adults with Fabry Disease

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Fabry’s disease
    Investigated Drugs:
    • Pegunigalsidase Alfa
    Belgium Czechia Denmark Italy Norway
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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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