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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Efficacy and Safety Evaluation of Odevixibat in Pediatric Biliary Atresia Post-Kasai Hepatoportoenterostomy: A Double-Blind, Randomized, Placebo-Controlled Trial

    Not recruiting

    3 1
    Investigated Drugs:
    • Odevixibat
    Belgium France Germany Hungary Italy The Netherlands +2

  • Study of ilofotase alfa effects on biomarkers in adults with hypophosphatasia

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Hypophosphatasia
    Investigated Drugs:
    • Ilofotase Alfa
    Germany

  • A study looking at the safety of switching from emicizumab to denecimig in adults and adolescents with haemophilia A with or without inhibitors

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Haemophilia A with anti factor VIII
    • Factor VIII deficiency
    Austria Belgium France Germany Italy Spain

  • A Study of ALN-6400 Safety and Effectiveness in Adults with Hereditary Hemorrhagic Telangiectasia

    Not recruiting

    1 1
    Investigated Drugs:
    • RNA, (UM-SP-(2′-DEOXY-2′-FLUORO)A-SP-GM-AM-AM-(2′-DEOXY-2′-FLUORO)A-CM-UM-CM-AM-UM-AM-GM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)A-UM-UM-GM-CM-AM-SP-CM-SP-AM), COMPLEX WITH RNA (UM-SP-GM-SP-CM-AM-AM-UM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-UM-GM-AM-GM-UM-UM-UM-CM-UM-AM) 3′-[[(2S,4R)-1-[29-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-14,14-BIS[[3-[[3-[[5-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]PROPYL]AMINO]-3-OXOPROPOXY]METHYL]-1,12,19,25-TETRAOXO-16-OXA-13,20,24-TRIAZANONACOS-1-YL]-4-HYDROXY-2-PYRROLIDINYL]METHYL HYDROGEN PHOSPHATE] (1:1)
    France Germany Spain

  • A Study to Evaluate the Safety of Olipudase Alfa in Children and Adults with Niemann-Pick Disease Who Completed Previous Treatment Studies

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Niemann-Pick disease
    France

  • Study of Navenibart to prevent attacks in adults and adolescents with Hereditary Angioedema

    Not recruiting

    3 1
    Investigated Diseases:
    • Hereditary angioedema with C1 esterase inhibitor deficiency
    • Hereditary angioedema
    Investigated Drugs:
    • NAVENIBART
    Austria Bulgaria Czechia France Germany Hungary +5
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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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