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Factor IX deficiency

  • A study on the long-term safety and effectiveness of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B

    Recruiting

    3 1 1
    Investigated Diseases:
    • Factor IX deficiency
    • Factor VIII deficiency
    Investigated Drugs:
    • Fidanacogene Elaparvovec
    • Giroctocogene Fitelparvovec
    France Germany Greece Italy Spain Sweden

  • Study on the Safety and Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Adults with Severe or Moderately Severe Hemophilia B and AAV5 Antibodies

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor IX deficiency
    Investigated Drugs:
    • Etranacogene Dezaparvovec
    Bulgaria Poland

  • Study on the Safety and Effectiveness of REGV131 and LNP1265 for Adults with Hemophilia B

    Recruiting

    2 1 1
    Investigated Drugs:
    • Adeno-Associated Virus Vector Serotype 8 Containing The Human F9 Gene
    • Messenger Rna Encoding Cas9
    • Single Guide Rna Containing A Sequence Complementary To Human Alb Locus Gene, Intron 1, Target Region
    France Germany Italy Spain

  • Study on Long-Term Safety and Effects of Marstacimab for Patients with Severe Hemophilia A or B, With or Without Inhibitors

    Recruiting

    3 1 1
    Investigated Diseases:
    • Factor IX deficiency
    • Factor VIII deficiency
    Investigated Drugs:
    • Marstacimab
    Austria Croatia Czechia Denmark France Germany +3

  • Study on Marstacimab for Children with Severe Hemophilia A or B, With or Without Inhibitors

    Recruiting

    3 1 1
    Investigated Drugs:
    • Marstacimab
    Austria Czechia Denmark France Germany Italy +2

  • Study of Etranacogene Dezaparvovec (CSL222) in Adolescent Males with Severe or Moderately Severe Hemophilia B

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Factor IX deficiency
    Investigated Drugs:
    • Etranacogene Dezaparvovec
    Austria Belgium France Spain
1 2
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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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