An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
IX-TEND 4001
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
2 other identifiers
observational
500
7 countries
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2023
Longer than P75 for all trials
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 15, 2023
CompletedFirst Submitted
Initial submission to the registry
August 15, 2023
CompletedFirst Posted
Study publicly available on registry
August 24, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2043
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 1, 2043
February 19, 2026
February 1, 2026
20.1 years
August 15, 2023
February 18, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Bleeding Rate (all bleeds) - HEMGENIX Cohort
Annualised Bleeding Rate (ABR) for all bleeds
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 Outcomes (15)
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.
Zero bleeds - HEMGENIX Cohort
During follow-up after HEMGENIX treatment, up to 180 months
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.
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.
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.
- +10 more secondary outcomes
Study Arms (2)
HEMGENIX
Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
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.
Interventions
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.
Eligibility Criteria
Patients with hemophilia B who have been treated with HEMGENIX and are enrolled in either the sponsor's study or the ATHN Transcends study NCT04398628 (Hemophilia Cohort, Gene Therapy Outcomes Arm), and patients who receive routine prophylaxis treatment with FIX replacement therapy that are enrolled in the ATHN Transcends study (Hemophilia Cohort, Natural History Arm), or a similar registry. All patients will provide signed informed consent required for participation.
You may qualify if:
- 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.
You may not qualify if:
- HEMGENIX Cohort:
- \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CSL Behringlead
Study Sites (12)
American Thrombosis and Hemostasis Network
Rochester, New York, 14604, United States
Medical University Vienna
Vienna, Austria
Aarhus Universitetshospital
Århus N, Denmark
Centre Hospitalier Universitaire de Brest / CHU Morvan
Brest, France
Centre Régional de Traitement de l'Hémophilie
Nantes, France
CHU Nancy - Hôpital Brabois
Vandœuvre-lès-Nancy, France
Klinik für Angiologie/ Hämostaseologie
Berlin, Germany
University of Clinic Bonn
Bonn, Germany
Klinikum der Johann-Wolfgang Goethe Universitaet
Frankfurt, Germany
Hannover Medical School
Hanover, Germany
Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
Vigo, Spain
University Hospital Bern Inselspital
Bern, Switzerland
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Global Program Director
CSL Behring
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 15, 2023
First Posted
August 24, 2023
Study Start
June 15, 2023
Primary Completion (Estimated)
August 1, 2043
Study Completion (Estimated)
August 1, 2043
Last Updated
February 19, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- Time Frame
- IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
- Access Criteria
- Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee. An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee. The requesting party must execute an appropriate data sharing agreement before IPD will be made available.
CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.