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
22
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
061002
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