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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on Bumetanide for Improving Cognitive Function in Children and Adolescents with Down Syndrome

    Recruiting

    2 1 1
    Investigated Diseases:
    • Trisomy 21
    Investigated Drugs:
    • BUMETANIDE
    Italy

  • Study on the Use of Epinephrine for Newborns with Cardiac Arrest at Birth

    Recruiting

    4 1 1 1
    Investigated Diseases:
    • Cardiac arrest
    Investigated Drugs:
    • Epinephrine
    France

  • Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Sickle cell anaemia
    Investigated Drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Busulfan
    Germany Italy

  • Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Thalassaemia
    Investigated Drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Filgrastim
    Germany Italy

  • Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

    Recruiting

    3 1 1
    Investigated Diseases:
    • Angelman’s syndrome
    Investigated Drugs:
    • Calcium Chloride
    • DISODIUM PHOSPHATE
    • Glucose, Anhydrous
    France Germany Spain

  • Study on Long-Term Safety of AAVAnc80-hOTOF Gene Therapy for Patients with Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

    Recruiting

    4 1 1
    Investigated Diseases:
    • Deafness
    Investigated Drugs:
    • Aavanc80-3’Hotof
    • Aavanc80-5’Hotof
    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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