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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis I
    Investigated Drugs:
    • Autologous Cd34+ Haematopoietic Stem And Progenitor Cells Genetically Modified With The Lentiviral Vector Idua Lv, Encoding For The Alpha-L-Iduronidase Cdna
    • Laronidase
    • Plerixafor
    Italy The Netherlands

  • Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

    Not recruiting

    2 1 1
    Investigated Drugs:
    • Human Alpha-1-Proteinase Inhibitor Immunoglobulin G Fusion Protein, Recombinant
    • Human Alpha1-Proteinase Inhibitor
    Denmark Ireland Poland Spain Sweden

  • Study on the Effects of Fidrisertib for Treating Fibrodysplasia Ossificans Progressiva in Children and Adults

    Not recruiting

    2 1
    Investigated Diseases:
    • Fibrodysplasia ossificans progressiva
    Investigated Drugs:
    • (R)‑Tetrahydrofuran‑3‑Yl 4‑(6‑(5‑(4‑Ethoxy‑1‑Isopropylpiperidin‑4‑Yl)Pyridin-2-Yl)Pyrrolo[1,2-B]Pyridazin-4-Yl)Piperazine-1-Carboxylate Sesquisuccinate
    Belgium France Germany Italy The Netherlands Portugal +2

  • 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 Effects of EryDex (Dexamethasone Sodium Phosphate) for Children Aged 6-9 with Ataxia Telangiectasia

    Not recruiting

    3 1
    Investigated Diseases:
    • Ataxia telangiectasia
    Investigated Drugs:
    • Dexamethasone Sodium Phosphate
    Belgium Denmark Germany Italy Norway Poland +1

  • 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
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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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