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

NCT06008938 · tracked via the Priya Life Science Germany tracker

Sponsor

CSL Behring

Phase

Observational

Started

2023-06-15

Last updated

2026-02-19

Condition(s) studied

Hemophilia B

Investigational drug(s) / intervention(s)

HEMGENIXFactor IX (FIX)

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.

Factor IX (FIX): FIX prophylaxis therapy

Study 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

Sex

ALL

Min age

18 Years

Max age

—

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.

Primary outcome measure(s)

Bleeding Rate (all bleeds) - HEMGENIX Cohort — 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.
Annualised Bleeding Rate (ABR) for all bleeds

Trial sites (12)

Facility	City	Region	Status
American Thrombosis and Hemostasis Network	Rochester	New York	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

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Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) Enrolling by invitation

Other trials for the same condition

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B Recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B Active, not recruiting

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy Active, not recruiting

German Pediatric Hemophilia Research Database Recruiting

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 Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06008938 on ClinicalTrials.gov ↗ ← All trials in Germany