BO41423 Emicizumab in Haemophilia A without FVIII Inhibitors

• Research type

  Research Study

• Full title

  A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB IN PATIENTS WITH MILD OR MODERATE HEMOPHILIA A WITHOUT FVIII INHIBITORS

• IRAS ID

  269459

• Contact name

  Peter Collins

• Contact email

  Peter.Collins@wales.nhs.uk

• Sponsor organisation

  F Hoffmann-La Roche Ltd

• Eudract number

  2019-002179-32

• Duration of Study in the UK

  2 years, 0 months, 0 days

• Research summary

  Haemophilia A is an X-linked recessive bleeding disorder that occurs in approximately
  1 in 5000 live male births.Patients with haemophilia A have a deficiency or absence of blood coagulation factor VIII(FVIII),an essential component of the intrinsic pathway in the coagulation cascade.

  Approximately 63% of people with haemophilia A have a moderate(19%)or severe(44%)form,leading to frequent bleeding events with the sequelae of musculoskeletal complications.
  Emicizumab is a recombinant, humanized, bispecific, immunoglobulin G4 monoclonal antibody that binds with moderate affinity to activated factor IX and factor X, mimicking the co-factor function of FVIII. In patients with haemophilia A, haemostasis can be restored irrespective of the presence of FVIII inhibitors, as emicizumab shares no sequence homology with FVIII.In addition, emicizumab offers the possibility of SC administration, removing the need for venous access.Finally, because of the pharmacokinetic and pharmacodynamic properties of this antibody, use of emicizumab enables dosing once a week, every 2 weeks, or every 4 weeks.
  Currently available experience with emicizumab in humans includes data from several studies. Based on the Phase III program, emicizumab gained approval in many countries and is indicated for routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in adults and children with haemophilia A with or without FVIII inhibitors and can be used in all age groups. In the European Union, the label is restricted to patients with severe haemophilia A

  The aim of this study is to investigate emicizumab prophylaxis (evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab) in patients with mild or moderate haemophilia A without inhibitors against FVIII whose bleeding phenotype warrants prophylactic treatment.
  Approx. 50 patients of all ages will be enrolled globally with 6 patients from 3 sites in the UK.

  The study is sponsored by F. Hoffman La Roche
  Research Summary; Version 1.0 dated 26 June 2019

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  19/SW/0153

• Date of REC Opinion

  9 Oct 2019

• REC opinion

  Further Information Favourable Opinion