Observational study on the effectiveness and safety of HEMGENIX in hemophilia B patients
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
This study is testing how well and safely HEMGENIX works for people with hemophilia B who are also getting Factor IX treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | CSL Behring Industry-sponsored |
| Locations | 12 sites (Rochester, New York and 11 other locations) |
| Trial ID | NCT06008938 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate both the short and long-term effectiveness and safety of HEMGENIX in patients diagnosed with hemophilia B. It will include a cohort of patients receiving Factor IX (FIX) prophylaxis to compare and interpret the efficacy and safety outcomes of HEMGENIX. Participants will be monitored for their response to treatment and any adverse effects over time, providing valuable data on the real-world application of HEMGENIX.
Who should consider this trial
Good fit: Ideal candidates include adult patients with hemophilia B who have received HEMGENIX treatment or are currently on FIX prophylaxis.
Not a fit: Patients who have previously been treated with etranacogene dezaparvovec in a clinical trial will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide critical insights into the effectiveness and safety of HEMGENIX, potentially improving treatment options for patients with hemophilia B.
How similar studies have performed: Other studies have shown promising results with similar gene therapies for hemophilia, suggesting potential for success in this observational study.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Rochester, New York and 11 other locations
- American Thrombosis and Hemostasis Network — Rochester, New York, United States (Recruiting)
- Medical University Vienna — Vienna, Austria (Recruiting)
- Aarhus Universitetshospital — Århus N, Denmark (Recruiting)
- Centre Hospitalier Universitaire de Brest / CHU Morvan — Brest, France (Recruiting)
- Centre Régional de Traitement de l'Hémophilie — Nantes, France (Recruiting)
- CHU Nancy - Hôpital Brabois — Vandœuvre-lès-Nancy, France (Recruiting)
- Klinik für Angiologie/ Hämostaseologie — Berlin, Germany (Recruiting)
- University of Clinic Bonn — Bonn, Germany (Recruiting)
- Klinikum der Johann-Wolfgang Goethe Universitaet — Frankfurt, Germany (Recruiting)
- Hannover Medical School — Hanover, Germany (Recruiting)
- Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez — Vigo, Spain (Recruiting)
- University Hospital Bern Inselspital — Bern, Switzerland (Recruiting)
Study contacts
- Study coordinator: Trial Registration Coordinator
- Email: clinicaltrials@cslbehring.com
- Phone: +1 610-878-4697
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.