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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study of lidocaine to reduce opioid use in patients with sickle cell disease experiencing severe pain crisis

    Recruiting

    3 1 1
    Investigated Drugs:
    • Lidocaine Hydrochloride
    • Sodium Chloride
    France

  • A Study of Gene Replacement Therapy for Children with CTNNB1 Syndrome Using Adeno-Associated Viral Vector Serotype 9 Containing the Human CTNNB1 Gene

    Recruiting

    2 1 1
    Investigated Diseases:
    • Neurodevelopmental disorder
    Investigated Drugs:
    • ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 CONTAINING THE HUMAN CTNNB1 GENE
    Slovenia

  • Title: Evaluating low-dose prednisone with standard therapy to improve heart function in patients with cardiac manifestations of Fabry disease

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Prednisone
    Poland

  • Study of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)

    Recruiting

    2 1 1
    Investigated Drugs:
    • Apitegromab
    Belgium France Italy The Netherlands Spain

  • Study of alpelisib in children and adults with PIK3CA-Related Overgrowth Spectrum (PROS)

    Recruiting

    2 1 1
    Investigated Diseases:
    • PIK3CA related overgrowth spectrum
    Investigated Drugs:
    • Alpelisib
    Austria Belgium France Germany Italy The Netherlands +1

  • Study of Elsunersen in Children with SCN2A Developmental and Epileptic Encephalopathy to Reduce Seizures

    Recruiting

    3 1 1
    Investigated Diseases:
    • Epileptic encephalopathy
    Investigated Drugs:
    • 5′-MOEMC-(SP)-MOEMC-(P)-MOEA-(P)-MOEMC-(P)-MOEG-(P)-MOEA-(P)-DMC-(SP)-DA-(SP)-DT-(SP)-DA-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DMC-(SP)-MOET-(P)-MOEA-(SP)-MOEMC-(SP)-MOEA 3′
    Germany Italy
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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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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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