BH30071 Prophylactic Emicizumab vs no Prophylaxis in Haemophilia A

• Research type

  Research Study

• Full title

  A RANDOMIZED, MULTICENTER, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF PROPHYLACTIC EMICIZUMAB VERSUS NO PROPHYLAXIS IN HEMOPHILIA A PATIENTS WITHOUT INHIBITORS

• IRAS ID

  202447

• Contact name

  Samya Gwen Obaji

• Contact email

  obajiSG1@cardiff.ac.uk

• Sponsor organisation

  F Hoffmann-La Roche Ltd

• Eudract number

  2016-000072-17

• Duration of Study in the UK

  1 years, 9 months, 1 days

• Research summary

  Summary of Research
  Haemophilia A is a bleeding disorder resulting from inherited deficiency/absence of blood coagulation factor VIII (FVIII). These patients have a lifelong bleeding tendency manifesting as easy bruising; prolonged bleeding; spontaneous bleeding typically into joints and intracranial haemorrhage. Haemophilia is classified into mild, moderate, and severe based on FVIII activity, and approximately half of patients have severe (<1% normal FVIII activity) form leading to frequent bleeding events with complications such as chronic joint disease, neurocognitive defects, or even death. These disease-related issues have a significant impact on the quality of life of adolescents and adults with haemophilia.\n\nRoutine FVIII replacement to prevent bleeds (prophylaxis) markedly reduces bleeding events and complications, and in children it is highly desired to start prophylaxis early in life. Although effective, this treatment is associated with a significant burden of infusion factor 3 times or more weekly (typically in the morning), which in itself impacts on quality of life. \n\nEmicizumab is an engineered antibody that mimics the function of FVIII. It is administered subcutaneously (SC), removing the need for venous access, and due to the pharmacokinetic properties of this antibody, the dosing interval is extended to once weekly or even less frequently. This novel compound has potential to dramatically change the treatment of haemophilia A patients with or without FVIII inhibitors who are in need of effective, safe and effective prophylactic therapy that is not associated with significant burden.\n\nThe primary objective for this study is to evaluate the efficacy of prophylactic emicizumab compared with no prophylaxis in patients with haemophilia A without inhibitors \n\nIn the UK inclusion of 8 patients across 4 sites is anticipated.\n

  Summary of Results
  The lay summary of study results were posted on the for patients website (published Jan 2021) Please see link provided below:-

  https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbSgv9uUS1wsd00iFWSZyptH89w1tcscJeKmImtXTgMdpxxTV-2Fu-2Ft3o5a6nKTeF-2FdrvvloupZbJGvif2JsdnPDUN18f6XRC-2BRT6PxayjErBmxGBgKhxxuRDQQvvmeRYiCg-2BsGQ-2BUikGHlZLwlrYgIVWY-3DERE4_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YKjAiTnl4jBv4BrOilo5LaMWxTay86arBWVZ0g4EDxiHQMtAyxh1Y1EfFso56-2B8tt0mBxH2QSx8ZkjoDTlQLld4IypzUnskzQwW-2BlWnt7m5tN-2FXxXB4PGgAICLRqVFFmwe7z-2FwgBLqOqDLpatQ2A6cFxdMf6hiKxYt4iNfQRg6DIMPgmJfUyfNw7vyzhi3fzyo-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7C5197f53a3eb74becbddc08dafdf04580%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638101505638025385%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=yoyIZQ%2FoZ0hz%2BOOnifsIp791Uxa%2FXTZG4CQDFhi0x6c%3D&reserved=0

  No further results will be provided.

• REC name

  Wales REC 3

• REC reference

  16/WA/0137

• Date of REC Opinion

  30 Jun 2016

• REC opinion

  Further Information Favourable Opinion