A Phase 3 study of Recombinant Coagulation Factor rFVIIIFc-VWF-XTEN

• Research type

  Research Study

• Full title

  A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A

• IRAS ID

  264092

• Contact name

  Sarah Mangles

• Contact email

  Sarah.Mangles@hhft.nhs.uk

• Sponsor organisation

  Bioverativ Therapeutics Inc.

• Eudract number

  2019-002023-15

• Duration of Study in the UK

  1 years, 3 months, 23 days

• Research summary

  Summary of Research
  This is a multinational, multicenter, open-label Phase 3 study of the safety, efficacy, and Pharmacokinetics (PK) of intravenous (IV) BIVV001 in previously treated patients (PTPs) ≥12 years of age with severe haemophilia A. The study is comprised of 2 Arms: Arm A and Arm B.
  Participants currently on a prophylaxis treatment regimen with FVIII will enter Arm A and receive BIVV001 at a dose of 50 IU/kg IV every week (52 weeks in total). Participants currently on an on-demand treatment regimen will enter Arm B for 26 weeks of on-demand treatment of bleeding episodes with BIVV001 at a dose of 50 IU/kg IV on demand, then switch to receive BIVV001 at a dose of 50 IU/kg IV every week on a prophylaxis treatment regimen for 26 weeks.
  Following a washout period (at least 4 to 5 days, depending on current therapy), all participants (except those in the sequential PK subgroup of Arm A) will undergo abbreviated PK sampling after the first dose of BIVV001 (Baseline). Sixteen participants at selected sites will participate in the sequential PK subgroup of Arm A and will undergo more extensive PK sampling at Baseline and again at Week 26.
  Participants from any arm who undergo major surgery during the study will be included in the surgery subset.
  Approximately 150 participants will be enrolled to collect sufficient data to assess the safety, efficacy, and PK of IV BIVV001 in this population. Approximately 124 participants will be in Arm A, of which 16 participants will be included in the sequential PK subgroup. Approximately 26 participants will be in Arm B. For the surgery subset, a minimum of 10 major surgeries in at least 5 participants will be targeted to assess control and prevention of bleeding in the surgical setting.
  The study is being sponsored by Bioverativ Therapeutics Inc.

  Summary of research
  The lay summary of the study results is available on: https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbfd6HtMZuEBXrupKun9DZUABp09Fn8d-2BpNBz2EbnLmTe0W7aVjtLnxEpfAkaflfiz11jDcTjS66ZWBzPVg1bZ48-3DH4YS_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YL7aFUK0En0qW8Gjj-2B-2Fzle0-2BSlMZkzB2VX0Ri9tu-2FkYc1t2O3fXTFC-2Bxy8P-2F-2FTHt35BO3QokHxZlfGQQ9RDrCQd26f-2FwIvE3iAA3XenxgTinX1PuxEIBGdpGKnzsPWG9HNd0h8HYWZkBvYs4Nowrr-2BqLoNcMpwhhcEQFR7EuyW-2Fbg-3D-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7Cdad97409b40845faecc808db0398a7dc%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638107726410952177%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=VC2J2gclg%2FwRjgJ4rGwUT2Jg9NpcgZMCJQuIQPOJMFI%3D&reserved=0

• REC name

  Wales REC 1

• REC reference

  19/WA/0319

• Date of REC Opinion

  6 Jan 2020

• REC opinion

  Further Information Favourable Opinion