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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Intranasal remestemcel therapy for newborns with hypoxic‑ischemic brain injury: a phase II placebo‑controlled trial

    Not yet recruiting

    2 1
    Investigated Diseases:
    • Brain injury
    Investigated Drugs:
    • Remestemcel
    The Netherlands

  • Safety and Efficacy of TSRA-196 (rnaivt9315 and rnacs24757) in Adults with Lung or Liver Disease due to Alpha‑1 Antitrypsin Deficiency

    Not yet recruiting

    2 1 1
    Investigated Diseases:
    • Alpha-1 antitrypsin deficiency
    Ireland The Netherlands Sweden

  • Safety and efficacy of autologous CD34+ cells transduced with a vector encoding human NCF1 in children and adults with p47‑deficient chronic granulomatous disease

    Not yet recruiting

    2 1 1
    Investigated Diseases:
    • Chronic granulomatous disease
    Germany Spain

  • Methoxyflurane versus oral oxycodone hydrochloride and morphine sulfate for pain relief in sickle cell disease vaso‑occlusive crises

    Not yet recruiting

    3 1 1 1
    Investigated Diseases:
    • Sickle cell disease
    Investigated Drugs:
    • Morphine Sulfate
    • Oxycodone Hydrochloride
    France

  • Safety and Efficacy of VX-828, Deutivacaftor, and Tezacaftor in Adults with Cystic Fibrosis

    Not yet recruiting

    2 1 1
    Investigated Diseases:
    • Cystic fibrosis
    Belgium Czechia Denmark France Germany Ireland +5

  • BMN 333 Versus Vosoritide in Children With Achondroplasia

    Not yet recruiting

    4 1 1 1
    Investigated Diseases:
    • Osteochondrodysplasia
    Investigated Drugs:
    • Vosoritide
    Italy Poland Romania
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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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