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

Plain-language summary

This is not an interventional trial but an observational, post-authorization, long-term follow-up cohort study. It investigates the short- and long-term effectiveness and safety of HEMGENIX (etranacogene dezaparvovec) in patients with hemophilia B. A comparison cohort of hemophilia B patients treated with FIX prophylaxis is also included; the HEMGENIX cohort requires treatment with commercial HEMGENIX and signed informed consent within the specified time windows.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

CSL Behring

Intervention

HEMGENIX (Etranacogene dezaparvovec), Factor IX (FIX)

Start date

6/15/2023

Estimated completion

8/1/2043

Countries

United States, Austria, Denmark, France, Germany, Spain, Switzerland

Related genes

Locations

• Rochester, United States
• Vienna, Austria
• Århus N, Denmark
• Brest, France
• Nantes, France
• Vandœuvre-lès-Nancy, France
• Berlin, Germany
• Bonn, Germany
• Frankfurt, Germany
• Hanover, Germany
• Vigo, Spain
• Bern, Switzerland

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.