An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B

Status: Recruiting
Location: See all (9) locations...
Intervention Type: Genetic, Biological
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• HEMGENIX Cohort:

• \- Treatment with commercial HEMGENIX.

• \- Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.

• FIX Prophylaxis Cohort:

• \- Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.

Locations
United States
New York
American Thrombosis and Hemostasis Network
RECRUITING
Rochester
Other Locations
Austria
Medical University Vienna
RECRUITING
Vienna
Denmark
Aarhus Universitetshospital
RECRUITING
Århus N
France
Centre Hospitalier Universitaire de Brest / CHU Morvan
RECRUITING
Brest
Centre Régional de Traitement de l'Hémophilie
RECRUITING
Nantes
CHU Nancy - Hôpital Brabois
RECRUITING
Vandœuvre-lès-nancy
Germany
Klinik für Angiologie/ Hämostaseologie
RECRUITING
Berlin
Hannover Medical School
RECRUITING
Hanover
Switzerland
University Hospital Bern Inselspital
RECRUITING
Bern
Contact Information
Primary
Trial Registration Coordinator
clinicaltrials@cslbehring.com
+1 610-878-4697
Time Frame
Start Date: 2023-06-15
Estimated Completion Date: 2043-08-01
Participants
Target number of participants: 500
Treatments
HEMGENIX
Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
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.
Related Therapeutic Areas
Sponsors
Leads: CSL Behring

This content was sourced from clinicaltrials.gov

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