IX-TEND 4001: An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| HEMGENIX Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use. |
Genetic: 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.
Other Names:
|
| 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. |
Biological: Factor IX (FIX)
FIX prophylaxis therapy
|
Outcome Measures
Primary Outcome Measures
- 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
- 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
- Zero bleeds - HEMGENIX Cohort [During follow-up after HEMGENIX treatment, up to 180 months]
Number of patients with zero bleeds
- 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.
- 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)
- 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).
- 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).
- 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 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).
- 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 Serious Adverse Events (SAEs) - number [During 15 years of follow-up]
Incidence (number) of related SAEs
- Related SAEs - percent [During 15 years of follow-up]
Incidence (percent) of related SAEs
- Adverse Events of Special Interest (AESIs) - number [During 15 years of follow-up]
Incidence (number) of AESIs
- AESIs - percent [During 15 years of follow-up]
Incidence (percent) of AESIs
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- CSL Behring
Investigators
- Study Director: Global Program Director, CSL Behring
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CSL222_4001