Early Prophylaxis Immunologic Challenge (EPIC) Study

Brief Title

Early Prophylaxis Immunologic Challenge (EPIC) Study

Official 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

Brief Summary

      The purpose of the study was to assess if a once-weekly prophylactic regimen of 25 IU/kg
      ADVATE started at or before 1 year of age and before the onset of a severe bleeding phenotype
      (ie, joint bleeding), together with the minimization of immunological danger signals, can
      reduce the incidence rate of inhibitor formation in PUPs with severe and moderately severe
      hemophilia A.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Number of Participants With Severe and Moderately Severe Hemophilia A (FVIII ≤ 2%) With Factor VIII (FVIII) Inhibitor Formation Within the First 50 Exposure Days to ADVATE

Secondary Outcome

 Number of Participants With Severe Hemophilia A (FVIII ≤ 1%) With Factor VIII (FVIII) Inhibitor Formation Within the First 50 Exposure Days to ADVATE

Condition

Hemophilia A

Intervention

Recombinant antihemophilic factor, plasma/albumin-free method (rAHF-PFM)

Study Arms / Comparison Groups

 ADVATE - Prophylactic Regimen

Description:  Weekly infusions of ADVATE. Study visits (physical examination, lab tests including FVIII inhibitor tests) every week during the first 10 exposure days (EDs), every 5 weeks during the next 10 EDs and every 10 weeks thereafter.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

August 26, 2011

Completion Date

November 16, 2012

Primary Completion Date

November 16, 2012

Eligibility Criteria

        Inclusion Criteria:

          -  Participants with severe and moderately severe hemophilia A (FVIII ≤ 2%)

          -  Participants < 1 year of age

          -  Participants must have ≤ 3 exposure days (EDs) to any FVIII concentrate or
             FVIII-containing product used for treatment of minor bleeds (bleeds requiring no more
             than 2 infusions per event), or for preventative or precautionary infusions following
             possible injury

          -  Participants with prior circumcision are allowed to enroll only if bleeding issues
             related to circumcision were the cause for the original diagnosis of hemophilia A and
             no more than 2 EDs of FVIII treatment were required

          -  Adequate venous access (without need for central venous access device
             (CVAD)-placement) as determined by the physician

          -  Written informed consent from legally authorized representative(s)

        Exclusion Criteria:

          -  Life-threatening conditions (intracranial hemorrhage, severe trauma) or requirement
             for surgery at the time of enrollment

          -  Evidence of inhibitor ≥ 0.6 Bethesda Unit (BU) in Nijmegen-modified Bethesda Assay at
             study start (samples may be retested using lupus-insensitive inhibitor tests to reduce
             the number of false positive inhibitor test results)

          -  Inherited or acquired hemostatic defect other than hemophilia A

          -  Any clinically significant, chronic disease other than hemophilia A

          -  Known hypersensitivity to ADVATE or any of its constituents

          -  Any planned elective surgery that cannot be postponed until after the first 20 EDs

          -  Participation in the Hemophilia Inhibitor Previously Untreated Patient Study

          -  Application of red blood cell, platelet, or leukocyte concentrates, or plasma

          -  Administration of any medication affecting coagulation or platelet function

          -  Systemic administration of any immunomodulatory drug (eg, chemotherapy, intravenous
             glucocorticoids)

          -  Participation in another clinical study involving an investigational product (IP) or
             device within 30 days prior to study enrollment or during the course of this study

Gender

Male

Ages

N/A - 1 Year

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

Austria

Location Countries

Austria

Administrative Informations

NCT ID

NCT01376700

Organization ID

Secondary IDs

2011-000410-18

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Collaborators

 Baxter Innovations GmbH

Study Sponsor

Study Director, Study Director, Takeda

Verification Date

April 2021