WP45338 – A Study of Emicizumab Prophylaxis in Patients with Type 3 Von Willebrand Disease

• Research type

  Research Study

• Full title

  A PHASE III, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH TYPE 3 VON WILLEBRAND DISEASE

• IRAS ID

  1011252

• Contact name

  Deepak Pardhi

• Contact email

  uk.dra@roche.com

• Sponsor organisation

  F. Hoffmann-La Roche Ltd

• Eudract number

  2024-515622-80

• Research summary

  This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 2 years and above, diagnosed with Type 3 VWD.

  Von Willebrand disease (VWD) is the most common autosomal inherited bleeding disorder, classified into Types 1, 2, and 3. Type 3 VWD is the most severe form of VWD, an autosomal recessive, extremely rare disorder.

  Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arms A and B), while participants on prior SOC prophylactic therapy (Arm C) will be assessed via intrapatient analysis with data from the preceding non-interventional study (NIS; WP45335).

  Emicizumab is approved to prevent bleeding in patients with hemophilia A with and without factor VIII (FVIII) inhibitors of all ages. However, emicizumab is not approved for Type 3 VWD and it is currently considered an experimental drug in this particular group of people.
  About 66 people with Type 3 VWD will take part in this study and the total duration of study participation for an individual (following screening) is expected to be approximately 2.5 years, or longer depending on duration of recruitment for the study.

  11 patients will be recruited across 4 UK sites.
  
  The study is sponsored by F. Hoffmann- La Roche
  
  Research Summary; Version Number 1 dated 17th October 2024

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  25/EM/0042

• Date of REC Opinion

  15 Apr 2025

• REC opinion

  Further Information Favourable Opinion