Evaluating the effectiveness and safety of FVIII concentrates in young haemophilia A 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 international observational study aims to assess the real-life effectiveness, safety, and utilization patterns of Octapharma's FVIII concentrates—Nuwiq, Octanate, and Wilate—in previously untreated and minimally treated patients with severe haemophilia A. The study focuses on young children and patients with limited exposure to FVIII, gathering data on treatment effectiveness and safety, particularly concerning inhibitor development. By collecting real-world evidence, the study seeks to enhance understanding of treatment protocols and inform future clinical practices.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Male and female patients of any age and ethnicity
* Severe haemophilia A (FVIII:C\<1%)
* Decision to prescribe Octapharma's FVIII concentrate before enrollment into the study
* Either
* No previous treatment with FVIII concentrates or other blood products containing FVIII (PUPs) OR
* Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood products containing FVIII (MTPs), if
* data are available on all previous treatment, AND
* they did not develop an inhibitor at any time point, OR
* they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate (in the presence or absence of emicizumab).
* Voluntarily given, fully informed written and signed consent obtained before any study-related data documentation is conducted (obtained from the patient's parent/legal guardian)

Exclusion Criteria:

* Diagnosis with a coagulation disorder other than haemophilia A
* Concomitant treatment with any systemic immunosuppressive drug
* Participation in an interventional clinical trial during the time period evaluated
* Participation in another non-interventional study of Octapharma

Where it's running

• University of Miami Miller School of Medicine
  Miami, Florida, United States Recruiting
• Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
  Minsk, Belarus Recruiting
• Hôpital Universitaire des Enfants Reine Fabiola
  Brussels, Belgium Recruiting
• Cliniques Universitaires Saint-Luc
  Brussels, Belgium Recruiting
• Cliniques Universitaires Saint-Luc
  Brussels, Belgium Recruiting
• University Hospital Centre Zagreb
  Zagreb, Croatia Recruiting

+9 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Cristina Solomon, MD
• Email: Cristina.Solomon@octapharma.com
• Phone: +41 79 585 90 42

Similar studies

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

• Denecimig (Mim8) for adults with acquired Haemophilia A

  This study tests whether denecimig can be safe and helpful for adults newly diagnosed with acquired Haemophilia A.

  Recruiting Phase 3 Novo Nordisk A/S · Iowa City, Iowa and 82 other locations · NCT07830732

• Surgical Use of Efanoctocog alfa in Hemophilia A Patients

  This observational study looks at how efanesoctocog alfa (Altuvoct) is used around surgery in people with hemophilia A to see if it helps control bleeding.

  Recruiting Hospices Civils de Lyon · Bron · NCT07723560

• SCT800 for preventing and treating bleeds in previously untreated severe hemophilia A patients

  This test tries SCT800, a recombinant factor VIII, as regular prevention and to treat bleeds in male patients with severe hemophilia A who haven't had prior factor VIII treatment.

  Recruiting Phase 3 Sinocelltech Ltd. · Beijing, Beijing Municipality · NCT07437404

• Different doses of Inno8 for adults with haemophilia A.

  This trial will test different doses of the experimental medicine Inno8 in adults with haemophilia A to see if it is safe and how the body handles it.

  Recruiting at 25 sites Phase 1 Novo Nordisk A/S · Aurora, Colorado and 32 other locations · NCT07220564

• Evaluating synovitis in adults with haemophilia A

  This study looks at how often adults with hemophilia A experience joint inflammation caused by bleeding and how it affects their joint health.

  Recruiting University Hospital, Bonn · Bonn, North Rhine-Westphalia · NCT06352216

• Survey on the success of individualized treatment for haemophilia A

  This study is testing whether a personalized high-dose treatment can help people with haemophilia A who have developed inhibitors against factor VIII and if certain lab tests can predict how well this treatment will work.

  Recruiting Haemophilia Centre Rhine Main · Frankfurt am Main, Hesse · NCT02207894

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.