Recruiting

HEMGENIX®

Sponsor:

CSL Behring

Code:

NCT06008938

Conditions

Hemophilia B

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

HEMGENIX

Factor IX (FIX)

Study Details

Brief 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.

Conditions

Hemophilia B

Study ID

NCT06008938

Start date

Jun 15, 2023

Status verified date

Feb, 2026

Completion date

Aug 1, 2043

Anticipated

Primary completion date

Aug 1, 2043

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

500 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

HEMGENIX

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)

FIX prophylaxis therapy

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 and Locations

Central contacts

Trial Registration Coordinator

+1 610-878-4697clinicaltrials@cslbehring.com

Locations

American Thrombosis and Hemostasis Network

Recruiting

Rochester, New York, United States, 14604

More Information

Sponsor

CSL Behring

Last update posted

Feb 19, 2026

Last verified

Feb, 2026

Keywords

  • Hemophilia B
  • HEMGENIX

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by CSL Behring on 2026-02-19.