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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study of RLY-2608 for Adults and Children with PIK3CA-Related Overgrowth and Malformations

    Recruiting

    2 1 1
    Investigated Diseases:
    • Lymphatic malformation
    • PIK3CA-activated mutation
    • PIK3CA related overgrowth spectrum
    Investigated Drugs:
    • RLY-2608
    Belgium France Germany Ireland Italy Norway +1
  • Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor VIII deficiency
    Investigated Drugs:
    • Efanesoctocog Alfa
    Croatia Czechia France Germany Ireland Italy +2
  • Study on the Safety and Effects of CRD-4730 for Patients with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

    Recruiting

    2 1
    Investigated Diseases:
    • Ventricular tachycardia
    Investigated Drugs:
    • Crd-4730
    France Italy The Netherlands Spain
  • Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia

    Recruiting

    2 1 1
    Investigated Drugs:
    • ATUMELNANT
    Germany Italy
  • Study of N-Acetyl-L-Leucine in Patients with Ataxia-Telangiectasia: A Phase III Trial

    Recruiting

    3 1
    Investigated Diseases:
    • Ataxia telangiectasia
    Investigated Drugs:
    • L-Acetylleucine
    Germany Slovakia Spain
  • Study on the Effects of Pitolisant Hydrochloride for Patients with Prader-Willi Syndrome

    Recruiting

    3 1
    Investigated Diseases:
    • Prader-Willi syndrome
    Belgium Czechia Denmark France Germany Italy +4
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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.

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