An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in
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
| Condition(s) | Hemophilia B |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| 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. |
| Who can participate | 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. |
| Ages | 18 Years |
| Sex | All |
| Lead sponsor | CSL Behring |
| Locations | Rochester, New York, United States; Vienna, Austria; Århus N, Denmark; Brest, France; Nantes, France; Vandœuvre-lès-Nancy, France (+6 more sites) |
| Start date | 2023-06-15 |
| NCT ID | NCT06008938 |
| Official listing | https://clinicaltrials.gov/study/NCT06008938 |