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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety of LY3884961 for Patients with Type 1 Gaucher Disease

    Not yet recruiting

    1 1 1 1
    Investigated diseases:
    • Gaucher’s disease type I
    Investigated drugs:
    • Eculizumab
    • Sirolimus
    • Methylprednisolone
    Germany Spain

  • Study on Gene Therapy AAVAnc80-hOTOF for Patients with Sensorineural Hearing Loss from Otoferlin Gene Mutations

    Not yet recruiting

    2 1 1
    Investigated diseases:
    • Deafness
    Investigated drugs:
    • Aavanc80-3’Hotof
    • Aavanc80-5’Hotof
    Spain

  • Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    • Sickle cell anaemia
    Investigated drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Busulfan
    France Italy

  • Study on ABCB5+ MSCs for Recessive Dystrophic and Junctional Epidermolysis Bullosa Patients

    Not yet recruiting

    3 1 1
    Investigated drugs:
    • Allogeneic Skin-Derived Abcb5-Positive Dermal Mesenchymal Stromal Cells
    • Chloropyramine Hydrochloride
    • Chlorphenamine Maleate
    Austria Croatia Denmark France Germany Greece +6

  • Study on the Safety and Effects of Acetylcysteine Amide for Patients Aged 12 and Over with Hereditary Cystatin C Amyloid Angiopathy

    Not yet recruiting

    2 1 1
    Investigated diseases:
    • Cerebral amyloid angiopathy
    Investigated drugs:
    • Acetylcysteine Amide
    Iceland

  • A study looking at the safety of switching from emicizumab to denecimig in adults and adolescents with haemophilia A with or without inhibitors

    Not recruiting

    3 1 1
    Austria Belgium France Germany Italy Spain
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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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