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An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B | Clinical Trials | Clareo Health

RECRUITING

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B

NCT06008938•CSL Behring

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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.

Locations (12)

American Thrombosis and Hemostasis Network

Rochester, New York, 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

Key Dates

Start Date

June 15, 2023

Primary Completion Date

August 1, 2043

Completion Date

August 1, 2043

Last Updated

February 19, 2026

Enrollment

500

ESTIMATED participants

Conditions

Hemophilia B

Interventions

HEMGENIX

GENETIC

Factor IX (FIX)

BIOLOGICAL

Contacts

Trial Registration Coordinator

CONTACT

+1 610-878-4697 clinicaltrials@cslbehring.com

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Hemophilia B

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 19, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Liver Biopsy Following Gene Therapy For Hemophilia

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to &lt; 18 Years of Age) With Severe or Moderately Severe Hemophilia B

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Liver Biopsy Following Gene Therapy For Hemophilia

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B