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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety and Long-Term Effects of Efavirenz for Adults and Late-Onset Juvenile Niemann-Pick Type C Disease with Cognitive Impairment

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Niemann-Pick disease
    Investigated Drugs:
    • Aprepitant
    Spain

  • Study on the Effects of Amiloride and Sodium Chloride on Vascular Stiffness in Patients with Polycystic Kidney Disease

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Congenital cystic kidney disease
    Investigated Drugs:
    • Amiloride
    • Potato Starch
    • Sodium Chloride
    The Netherlands

  • Study on Carbetocin Nasal Spray for Reducing Uncontrollable Hunger in Patients with Prader-Willi Syndrome

    Not recruiting

    3 1
    Investigated Diseases:
    • Hyperphagia
    Investigated Drugs:
    • Carbetocin
    Belgium France Germany Italy Spain

  • Study of ION373 Given by Spinal Injection for Patients with Alexander Disease

    Not recruiting

    2 1
    Investigated Diseases:
    • Alexander disease
    Investigated Drugs:
    • 2′-O-(2-Methoxyethyl)-D-Ribose Antisense Oligonucleotide Targeting Glial Fibrillary Acidic Protein Messenger Ribonucleic Acid
    Italy The Netherlands

  • Study on the Safety and Feasibility of Oral Zinc Acetate Dihydrate for Patients with GNAO1-Related Disorders

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Neurodevelopmental disorder
    Investigated Drugs:
    • Zinc Acetate Dihydrate
    Germany

  • Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels

    Not recruiting

    3 1
    Investigated Diseases:
    • Familial hypertriglyceridaemia
    • Hyperchylomicronaemia
    Investigated Drugs:
    • Isis 678354 Sodium Salt
    Bulgaria Czechia Denmark Finland France Germany +9
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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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