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Gaucher’s disease type I

  • Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1 Using Drug Combination

    Not yet recruiting

    3 1 1 1
    Investigated Diseases:
    • Gaucher’s disease type I
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype S3 Containing Codon-Optimised Expression Cassette Encoding Human Beta-Glucocerebrosidase Variant
    • Methylprednisolone Hydrogen Succinate
    • Prednisolone
    Spain
  • Study on the Safety of LY3884961 for Patients with Type 1 Gaucher Disease

    Not yet recruiting

    1 1 1 1
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype 9 Expressing Codon-Optimized Human Gba Gene
    • Eculizumab
    • Methylprednisolone
    Germany Spain
  • Study of Eliglustat alone or with Imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Gaucher’s disease type I
    • Gaucher’s disease type III
    Investigated Drugs:
    • Eliglustat Tartrate
    • Imiglucerase
    France Italy Spain
  • Study on the Long-term Safety of FLT201 for Patients with Gaucher Disease Type 1

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Gaucher’s disease type I
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype S3 Containing Codon-Optimised Expression Cassette Encoding Human Beta-Glucocerebrosidase Variant
    Spain
  • Study of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease Type 3

    Not recruiting

    2 1 1 1
    Investigated Diseases:
    • Gaucher’s disease type I
    • Gaucher’s disease type III
    Investigated Drugs:
    • Imiglucerase
    • Venglustat
    Germany

Investigational Drugs in This Location

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Medical Conditions Being Studied in This Location

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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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