Early prophylaxis immunologic challenge (EPIC) study

• Research type

  Research Study

• Full title

  A Phase 3b clinical study to assess whether regular administration of ADVATE in the absence of immunological danger signals reduces the incidence rate of inhibitors in previously untreated patients with hemophilia A

• IRAS ID

  80850

• Contact name

  Elizabeth Chalmers

• Sponsor organisation

  Baxter Innovations GmbH

• Eudract number

  2011-000410-18

• Research summary

  This is a Phase 3b, prospective, historically controlled, single arm, international, multicenter study of ADVATE Antihaemophilic Factor in previously untreated patients (PUPs) with severe and moderately severe haemophilia A. The study is designed to assess whether regular administration of ADVATE at an early age and in the absence of immunological danger signals reduces the incidence rate of inhibitors in previously untreated patients with haemophilia A. The inhibitor rate will be compared to the rates for patients from historical cohorts, including the ADVATE PUP Study (Baxter clinical study 060103). The estimated number of study participants is 100, and the estimated number of global study sites is approximately 75. The overall duration of the study is 5 years from study initiation (i.e., first patient enrolled) to study completion (i.e., last patient last visit). The recruitment period is expected to be 3 years. The patient participation period is a maximum of 2 years from enrollment to subject completion (i.e., last study visit), unless prematurely discontinued. ADVATE will be administered by intravenous infusion at a dose of 25ñ5 IU/kg once per week (increasing to 2 or 3 times per week, if required) and continue throughout the first 20 exposure days (EDs). After 20 EDs, the weekly infusions should be continued for as long as possible following this early prophylaxis period. All tests performed during the study will be normal clinical procedures.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  11/ES/0011

• Date of REC Opinion

  16 Nov 2011

• REC opinion

  Further Information Favourable Opinion