An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

Part of paid clinical trials in Rochester, New York.

Sponsor
CSL Behring
Study ID
NCT06008938
Status
Recruiting
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Conditions

  • Hemophilia B

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • HEMGENIX — GENETIC
    HEMGENIX is a gene therapy medicinal product that aims to deliver a factor IX (FIX) gene expression cassette to the liver of patients with hemophilia B.
  • Factor IX (FIX) — BIOLOGICAL
    FIX prophylaxis therapy

Study Details

This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.

Key Dates

Start date
Jun 15, 2023
Status verified
Feb 2026
Primary completion
Aug 1, 2043
Completion
Aug 1, 2043

Study Design

Enrollment
500 participants (estimated)

Arms

  • Arm: HEMGENIX
    Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
  • Arm: FIX Prophylaxis
    Patients with hemophilia B on FIX prophylaxis and enrolled in American Thrombosis and Hemostasis Network (ATHN) Transcends (A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People with Non-Neoplastic Hematologic Disorders) Hemophilia Cohort, or a similar registry.

Primary Outcome Measure

Bleeding Rate (all bleeds) - HEMGENIX Cohort [ Time Frame: During the 52 weeks following stable FIX expression (6 to 18 months) in the follow-up period and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment. ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
American Thrombosis and Hemostasis NetworkRochesterNew York14604-

Find similar trials in Rochester, NY

By research site
American Thrombosis and Hemostasis Network· Rochester, NY

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