An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B (IX-TEND 4001)

CSL Behring

Status: Enrolling

Conditions: Hemophilia B

Treatments: Biological: Factor IX (FIX); Genetic: HEMGENIX

Study type: Observational

Funder types: Industry

Identifiers

NCT06008938

CSL222_4001

EUPAS106066 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 500 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

• HEMGENIX Cohort:
• - The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.

Trial design

500 participants in 2 patient groups

HEMGENIX

Description:

Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.

Treatment:

Genetic: HEMGENIX

FIX Prophylaxis

Description:

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.

Treatment:

Biological: Factor IX (FIX)