Observational study on the effectiveness and safety of HEMGENIX in hemophilia B patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to evaluate both the short and long-term effectiveness and safety of HEMGENIX in patients diagnosed with hemophilia B. It will include a cohort of patients receiving Factor IX (FIX) prophylaxis to compare and interpret the efficacy and safety outcomes of HEMGENIX. Participants will be monitored for their response to treatment and any adverse effects over time, providing valuable data on the real-world application of HEMGENIX.

Why it matters

Who can join: the full rules

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.

Where it's running

• American Thrombosis and Hemostasis Network
  Rochester, New York, United States 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

Study contacts

• Study coordinator: Trial Registration Coordinator
• Email: clinicaltrials@cslbehring.com
• Phone: +1 610-878-4697

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

  This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.

  Recruiting at 7 sites Phase 1/2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07644832

• Single-dose CSL222 gene therapy for adolescent males (12–17) with severe or moderately severe hemophilia B.

  This will give a single dose of CSL222 gene therapy to adolescent males with severe or moderately severe hemophilia B to see if it raises factor IX activity and reduces bleeding.

  Recruiting at 17 sites Phase 3 CSL Behring · Orange, California and 16 other locations · NCT07080905

• ATHNdataset registry for people with bleeding and clotting disorders

  This registry collects health, treatment, and patient-reported information from people with bleeding and clotting disorders seen at ATHN-affiliated centers to help improve care and outcomes.

  Recruiting American Thrombosis and Hemostasis Network · Hickory, North Carolina · NCT06820515

• Is synovial proliferation in haemophilia joints active or inactive?

  This project will test whether ultrasound, MRI and elastography can tell if subclinical synovial proliferation in people with severe haemophilia is currently active so clinicians can treat earlier.

  Recruiting UMC Utrecht · Utrecht · NCT06809972

• Observational study of blood clotting and bleeding disorders in Chinese patients

  This study looks at blood clotting and bleeding problems in Chinese patients to see how common they are and what factors might affect them.

  Recruiting Peking University People's Hospital · Beijing, Beijing Municipality and 4 other locations · NCT06727669

• Comparing ANB-002 to standard treatment for hemophilia B

  This study is testing a new treatment called ANB-002 to see if it works as well as the standard treatment for adults with hemophilia B who have low FIX activity and no FIX inhibitors.

  Recruiting at 11 sites Phase 3 Biocad · Chelyabinsk and 13 other locations · NCT06700096

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.