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Evaluating the effectiveness and safety of FVIII concentrates in young haemophilia A patients

Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW

Observational Octapharma · NCT03695978

This study is testing how well and safely a new treatment for hemophilia A works in young children who haven't had much treatment before.

Quick facts

Study typeObservational
Enrollment200 (estimated)
SexAll
SponsorOctapharma Industry-sponsored
Drugs / interventionsemicizumab
Locations55 sites (Miami, Florida and 54 other locations)
Trial IDNCT03695978 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include male and female patients of any age with severe haemophilia A who are either previously untreated or minimally treated with FVIII concentrates.

Not a fit: Patients with coagulation disorders other than haemophilia A or those who have had significant prior exposure to FVIII concentrates may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the optimal treatment strategies for young patients with haemophilia A, potentially improving their health outcomes.

How similar studies have performed: Other studies have shown success in evaluating FVIII concentrates in haemophilia A, but this study aims to provide additional real-world evidence in a specific patient population.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Miami, Florida and 54 other locations

+5 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Haemophilia A
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.