Hemophilia B
Conditions
Keywords
Hemophilia B, HEMGENIX
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.
Interventions
GENETICHEMGENIX
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.
BIOLOGICALFactor IX (FIX)
FIX prophylaxis therapy
Sponsors
CSL Behring
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Austria, Denmark, France, Germany, Spain, Switzerland, United States
Contacts
CONTACTTrial Registration Coordinator
STUDY_DIRECTORGlobal Program Director
CSL Behring
Outcome results
None listed