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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment

    Recruiting

    2 1 1 1
    Investigated Drugs:
    • AUTOLOGOUS HAEMATOPOIETIC STEM AND PROGENITOR CELL POPULATION CONTAINING CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR ENCODING THE TCIRG1 CDNA EX VIVO EXPANDED
    • Plerixafor
    • Thiotepa
    Italy

  • Study of NVD003 stem cell implant compared to bone graft for children with congenital pseudarthrosis of the tibia

    Recruiting

    3 1 1
    Investigated Drugs:
    • Autologous Adipose-Derived Stem Cells
    Belgium France Spain

  • Study of metformin for cancer prevention in adolescents and adults with Li-Fraumeni syndrome

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Metformin
    • Metformin Hydrochloride
    Germany

  • Study on the Effectiveness and Safety of Infigratinib for Children with Hypochondroplasia and Short Stature

    Recruiting

    4 1 1
    Investigated Diseases:
    • Hypochondroplasia
    Investigated Drugs:
    • Infigratinib
    • Placebo
    France Norway Portugal Spain Sweden

  • Study on [18F]MNI-659 to Track Huntington’s Disease Progression in Symptomatic and Pre-symptomatic Patients

    Recruiting

    3 1 1
    Investigated Drugs:
    • 2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE
    France

  • Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1

    Recruiting

    2 1 1
    Investigated Diseases:
    • Primary hyperoxaluria
    Investigated Drugs:
    • GRNA-001
    France Germany The Netherlands
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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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