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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety of DNL310 for Children with Hunter Syndrome

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis II
    Investigated Drugs:
    • Iduronate-2-Sulfatase Fused To A Fc Polypeptide That Binds To The Human Transferrin Receptor
    The Netherlands

  • Study on MAS825 for Patients with Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Autoinflammation with infantile enterocolitis
    • Immune-mediated enterocolitis
    Investigated Drugs:
    • Ciclosporin
    • Human Igg1 Monoclonal Antibody Against Human Il-1 Beta And Human Il-18
    Czechia France Italy Spain

  • Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Hereditary neuropathic amyloidosis
    • Cardiac amyloidosis
    Investigated Drugs:
    • Patisiran
    • Vutrisiran
    Belgium Bulgaria Cyprus Italy The Netherlands Portugal +1

  • Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven’t received treatment for 6 months

    Not recruiting

    3 1
    Investigated Diseases:
    • Fabry’s disease
    Investigated Drugs:
    • Venglustat
    Austria Denmark Finland France Germany Greece +4

  • Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

    Not recruiting

    4 1 1
    Investigated Drugs:
    • Leriglitazone
    France Germany Spain

  • Study on NVD-003 Bone Graft for Treating Congenital Pseudarthrosis of the Tibia in Children

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Congenital pseudarthrosis
    Investigated Drugs:
    • Autologous Adipose-Derived Stem Cells
    Belgium
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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.

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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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