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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease

    Recruiting

    3 1
    Investigated Diseases:
    • Sickle cell disease
    Investigated Drugs:
    • Etavopivat
    Belgium France Greece Italy The Netherlands Spain
  • Study on the Effectiveness of Oxybutynin for Children with Overactive Neurogenic Bladder Due to Spina Bifida

    Recruiting

    3 1 1
    Investigated Diseases:
    • Spina bifida
    Investigated Drugs:
    • OXYBUTYNIN HYDROCHLORIDE
    France
  • Study on the Effects of Full Spectrum Cannabis Extract (Dronabinol, Cannabidiol) for Patients with Hard-to-Treat Epilepsy in Tuberous Sclerosis Complex

    Recruiting

    3 1
    Investigated Diseases:
    • Tuberous sclerosis complex
    Investigated Drugs:
    • Dronabinol
    • Cannabidiol
    Spain
  • Study on the Effectiveness and Safety of Nizubaglustat in Patients with Niemann-Pick Type C, GM1, or GM2 Gangliosidosis

    Recruiting

    3 1
    Investigated Drugs:
    • NIZUBAGLUSTAT
    France Germany Italy Portugal Spain Sweden
  • Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

    Recruiting

    3 1 1
    Investigated Drugs:
    • Deucrictibant
    Austria Bulgaria France Germany Hungary Ireland +5
  • Study Comparing Metformin and Tolvaptan for Adults with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Congenital cystic kidney disease
    Investigated Drugs:
    • Metformin
    • Tolvaptan
    Italy
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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.

Explore the full ecosystem of clinical research in Europe.

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.

Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).

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