261202: BAX855 in children with severe haemophilia A

• Research type

  Research Study

• Full title

  A PHASE 3 PROSPECTIVE, UNCONTROLLED, MULTICENTER STUDY EVALUATING PHARMACOKINETICS, EFFICACY, SAFETY, AND IMMUNOGENICITY OF BAX 855 (PEGYLATED FULL-LENGTH RECOMBINANT FVIII) IN PREVIOUSLY TREATED PEDIATRIC PATIENTS WITH SEVERE HEMOPHILIA A

• IRAS ID

  158751

• Contact name

  Raina Liesner

• Contact email

  ri.liesner@gosh.nhs.uk

• Sponsor organisation

  Baxter Innovations GmbH

• Eudract number

  2014-000742-30

• Research summary

  Haemophilia is a serious and life-threatening disease if not treated. Current management of severe haemophilia A includes on-demand treatment for bleeding events and/or prophylaxis to prevent bleeding. Current prophylaxis treatments require infusions every other day, or every 2 to 3 days depending on individual patients. A new drug, BAX 855, is designed to work for longer, thus reducing the frequency of bleeding episodes and therefore improving patient convenience and compliance with therapy and thereby, improving overall health outcomes.

  About 60 children with haemophilia A aged less than 12 years old will participate in this study of BAX 855. The study will be conducted at specialist haematology centres in Europe, Asia, and the United States. Participation in this study will last for approximately 8 to 10 months depending on whether or not participants take part in the optional Pharmacokinteic (PK) assessments. During the main part of the study, participants will have 4 clinic/hospital visits and be contacted regularly by telephone. Participants will receive twice weekly treatment with 50 ±5 IU/kg of BAX 855 over a period of approximately 6 months.

  The main purpose of the study is to determine:
  • If BAX 855 is more effective in preventing and treating bleeding than other standard Factor VIII products in paediatric Haemophilia A patients
  • If BAX 855 is safe and well tolerated in paediatric Haemophilia A patients

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  14/LO/1536

• Date of REC Opinion

  22 Oct 2014

• REC opinion

  Further Information Favourable Opinion