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Clinical Trials in the USA / NCT06008938
Recruiting Observational

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

NCT06008938 · tracked via the Priya Life Science USA 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
No
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)

FacilityCityRegionStatus
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

More CSL Behring trials in the USA

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) Enrolling by invitation
Two Dose Levels of Privigen in Pediatric CIDP Recruiting
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis Recruiting
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis Active, not recruiting
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE) Recruiting

Other trials for the same condition

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) Enrolling by invitation
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
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
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency) 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
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 the USA