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

ConditionHemophilia B
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorCSL Behring

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

- 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.

- 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.

Disqualifiers

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

Trial population

Patients with hemophilia B who have been treated with HEMGENIX and are enrolled in either the sponsor's study or the ATHN Transcends study NCT04398628 (Hemophilia Cohort, Gene Therapy Outcomes Arm), and patients who receive routine prophylaxis treatment with FIX replacement therapy that are enrolled in the ATHN Transcends study (Hemophilia Cohort, Natural History Arm), or a similar registry. All patients will provide signed informed consent required for participation.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • 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/Vaccine

    FIX prophylaxis therapy

Treatment groups

500 Participants
are divided into 2 treatment groups
Group A: HEMGENIX1 intervention
Group B: FIX Prophylaxis1 intervention

Trial outcomes

Primary outcomes

1

Bleeding Rate (all bleeds) - HEMGENIX Cohort

Annualised Bleeding Rate (ABR) for all bleeds

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.

Secondary outcomes

1

Bleeding Rate (specified bleed types) - HEMGENIX Cohort

ABR for spontaneous, FIX-treated, traumatic, and joint bleeds

Time frame
During follow-up period (52 weeks following stable FIX expression [6 to 18 months]), and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
2

Zero bleeds - HEMGENIX Cohort

Number of patients with zero bleeds

Time frame
During follow-up after HEMGENIX treatment, up to 180 months
3

Correlation analysis - HEMGENIX Cohort

Correlation analysis of ABR as a function of mean FIX activity.

Time frame
Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.
4

Bleeding Rate - FIX Prophylaxis Cohort

ABR (total, spontaneous, FIX-treated, traumatic, and joint bleeds)

Time frame
In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

CSL Behring

Lead sponsor

Source ClinicalTrials.gov
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