Recruiting NCT06008938
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: HEMGENIX, Factor IX (FIX).
- Who it may be relevant to
- Registry conditions: Hemophilia B. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Austria, Denmark, France, Germany +2
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
Overview
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
- 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. - Biological Factor IX (FIX)
FIX prophylaxis therapy
Primary outcome measures
- Bleeding Rate (all bleeds) - HEMGENIX Cohort [Time frame: 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.]
Secondary outcome measures (12)
- Bleeding Rate (specified bleed types) - HEMGENIX Cohort [Time frame: 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.]
- Zero bleeds - HEMGENIX Cohort [Time frame: During follow-up after HEMGENIX treatment, up to 180 months]
- Correlation analysis - HEMGENIX Cohort [Time frame: Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.]
- Bleeding Rate - FIX Prophylaxis Cohort [Time frame: In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.]
- FIX activity - HEMGENIX Cohort [Time frame: 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.]
- Annualized consumption of FIX replacement therapy - HEMGENIX Cohort [Time frame: 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).]
- Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort [Time frame: At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.]
- Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort [Time frame: At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.]
- Target joints occurrence - HEMGENIX Cohort [Time frame: 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).]
- Target joints resolution - HEMGENIX Cohort [Time frame: During the follow-up period, up to 180 months]
- Target joints - FIX Prophylaxis Cohort [Time frame: From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.]
- Related Serious Adverse Events (SAEs) - number [Time frame: During 15 years of follow-up]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 4 centers
- Klinik für Angiologie/ Hämostaseologie — Berlin
- University of Clinic Bonn — Bonn
- Klinikum der Johann-Wolfgang Goethe Universitaet — Frankfurt
- Hannover Medical School — Hanover
France · 3 centers
- Centre Hospitalier Universitaire de Brest / CHU Morvan — Brest
- Centre Régional de Traitement de l'Hémophilie — Nantes
- CHU Nancy - Hôpital Brabois — Vandœuvre-lès-Nancy
United States · 1 center
- American Thrombosis and Hemostasis Network — Rochester
Austria · 1 center
- Medical University Vienna — Vienna
Denmark · 1 center
- Aarhus Universitetshospital — Århus N
Spain · 1 center
- Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez — Vigo
Switzerland · 1 center
- University Hospital Bern Inselspital — Bern
Identifiers
NCT: NCT06008938 · CSL222_4001 · EUPAS106066