An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
- Conditions
- Hemophilia B
- Interventions
- Biological: Factor IX (FIX)Genetic: HEMGENIX
- Registration Number
- NCT06008938
- Lead Sponsor
- CSL Behring
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 500
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.
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
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description FIX Prophylaxis Factor IX (FIX) 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. HEMGENIX HEMGENIX Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
- Primary Outcome Measures
Name Time Method Bleeding Rate (all bleeds) - HEMGENIX Cohort 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. Annualised Bleeding Rate (ABR) for all bleeds
- Secondary Outcome Measures
Name Time Method Zero bleeds - HEMGENIX Cohort During follow-up after HEMGENIX treatment, up to 180 months Number of patients with zero bleeds
Related Serious Adverse Events (SAEs) - number During 15 years of follow-up Incidence (number) of related SAEs
Adverse Events of Special Interest (AESIs) - number During 15 years of follow-up Incidence (number) of AESIs
Bleeding Rate (specified bleed types) - HEMGENIX Cohort 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. ABR for spontaneous, FIX-treated, traumatic, and joint bleeds
FIX activity - HEMGENIX Cohort Day 1, Week 6, and Months 6, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment. Mean uncontaminated endogenous FIX activity (%)
Annualized consumption of FIX replacement therapy - HEMGENIX Cohort During the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], & at Months 0 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180), & at yearly intervals (ie, at 0 to 1 yr, 1 to 2 yrs, etc, up to 14 to 15 yrs). Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).
Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment. Number and proportion of patients remaining free of previous continuous routine prophylaxis (FIX replacement therapy)
Target joints - FIX Prophylaxis Cohort From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180. The annualized occurrence of target joints
Related SAEs - percent During 15 years of follow-up Incidence (percent) of related SAEs
Correlation analysis - HEMGENIX Cohort Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment. Correlation analysis of ABR as a function of mean FIX activity.
Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180. Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).
AESIs - percent During 15 years of follow-up Incidence (percent) of AESIs
Bleeding Rate - FIX Prophylaxis Cohort In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months. ABR (total, spontaneous, FIX-treated, traumatic, and joint bleeds)
Target joints occurrence - HEMGENIX Cohort After HEMGENIX treatment, during the follow-up periods (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). The annualized occurrence of target joints (defined as 3 or more spontaneous bleeding events into a single joint within a consecutive 6-month period).
Target joints resolution - HEMGENIX Cohort During the follow-up period, up to 180 months The percentage of resolution of target joints (target resolution is defined as a recorded target joint with 2 or fewer spontaneous bleeding events within a consecutive 12-month period).
Trial Locations
- Locations (1)
American Thrombosis and Hemostasis Network
🇺🇸Rochester, New York, United States