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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • A Phase III study of NXT007 compared with simoctocog alfa prophylaxis in patients with Hemophilia A without inhibitors

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Simoctocog Alfa
    • Ro7589655
    Austria Denmark France Germany Hungary Italy +3

  • Evaluation of NXT007 versus Emicizumab Prophylaxis in Patients with Hemophilia A

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor VIII deficiency
    Investigated Drugs:
    • Emicizumab
    • Ro7589655
    Austria Belgium Denmark France Germany Hungary +5

  • A study to evaluate the effectiveness and safety of vamifeport in adults with hereditary hemochromatosis

    Recruiting

    2 1
    Investigated Drugs:
    • VAMIFEPORT TRIHYDROCHLORIDE
    Austria Belgium Czechia France Germany Ireland +5

  • A Phase 3 Study of Fenfluramine Hydrochloride for Safety and Efficacy in Patients with Rett Syndrome

    Recruiting

    3 1 1
    Investigated Drugs:
    • Fenfluramine Hydrochloride
    Belgium France Germany Hungary Italy Poland +1

  • Long‑Term Safety and Efficacy of Infigratinib in Children with Hypochondroplasia

    Recruiting

    2 1 1
    Investigated Drugs:
    • Infigratinib
    Norway Spain

  • A Phase 3 study to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome

    Recruiting

    3 1 1
    Investigated Diseases:
    • Angelman’s syndrome
    Investigated Drugs:
    • 2′-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Ube3A Antisense Transcript Rna
    Germany Italy Poland Spain
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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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