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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety and Effectiveness of CRISPR-Cas9 Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel

    Not recruiting

    3 1 1 1
    Investigated Drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Filgrastim
    Germany

  • Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis III
    Investigated Drugs:
    • N-Sulfoglucosamine Sulfohydrolase Fused To A Humanised Monoclonal Antibody Targeting Human Transferrin Receptor
    Germany

  • Study on the Effectiveness and Safety of Vosoritide for Children with Hypochondroplasia

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Hypochondroplasia
    Investigated Drugs:
    • Vosoritide
    France Germany Italy Spain

  • Study on Long-Term Safety of INBRX-101 for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Emphysema
    • Alpha-1 antitrypsin deficiency
    Investigated Drugs:
    • Human Alpha-1-Proteinase Inhibitor Immunoglobulin G Fusion Protein, Recombinant
    Denmark Ireland Poland Spain Sweden

  • Study on the Safety and Effects of Berotralstat for Children Aged 2 to 12 with Hereditary Angioedema (HAE)

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Hereditary angioedema
    Investigated Drugs:
    • Berotralstat
    Austria France Germany Italy Poland Spain

  • Study of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Ornithine transcarbamoylase deficiency
    Investigated Drugs:
    • Avalotcagene Ontaparvovec
    • Sodium Acetate (1-13C)
    • Prednisolone
    France Germany Italy The Netherlands Portugal Spain
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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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