Early Prophylaxis Immunologic Challenge (EPIC) Study

This study has been terminated.
(It became unlikely to achieve the study objective of 50% reduction over published inhibitor rates. The Data Monitoring Committee supported this decision.)
Sponsor:
Collaborator:
Baxter Innovations GmbH
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01376700
First received: June 17, 2011
Last updated: April 3, 2013
Last verified: April 2013
  Purpose

The purpose of the study is 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.


Condition Intervention Phase
Hemophilia A
Biological: Recombinant antihemophilic factor, plasma/albumin-free method (rAHF-PFM)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
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

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • Incidence of inhibitor formation in severe and moderately severe hemophilia A (FVIII <= 2%) within the first 50 exposure days to ADVATE [ Time Frame: 50 exposure days to ADVATE ] [ Designated as safety issue: Yes ]

Enrollment: 22
Study Start Date: August 2011
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Once-weekly prophylactic regimen
Weekly infusions of ADVATE. Study visits (physical examination, lab tests including FVIII inhibitor tests) will occur every week during the first 10 exposure days (EDs), every 5 weeks during the next 10 EDs and every 10 weeks thereafter.
Biological: Recombinant antihemophilic factor, plasma/albumin-free method (rAHF-PFM)
Intravenous infusion at a dose of 25 ± 5 IU/kg once per week. After 20 exposure days, the weekly infusions should be continued for as long as possible following the early prophylaxis period. If required by the clinical situation, dosing may be increased to twice weekly or even three times weekly after 20 exposure days, while keeping the low dose.
Other Name: ADVATE

  Eligibility

Ages Eligible for Study:   up to 1 Year
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects with severe and moderately severe hemophilia A (FVIII <= 2%); certain FVIII mutation types (e.g., large multi-domain deletions, nonsense mutations, insertions/deletions/inversions that result in a premature stop codon, intron 22 inversions) can be used to corroborate a severe hemophilia A phenotype when laboratory assays show FVIII levels >= 1% because of rounding errors or carryover effect from a previous FVIII administration; a central laboratory FVIII assay is required to confirm subject eligibility.
  • Subjects < 1 year of age
  • Subjects 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
  • Subjects 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01376700

  Show 62 Study Locations
Sponsors and Collaborators
Baxter Healthcare Corporation
Baxter Innovations GmbH
Investigators
Study Director: Armin Reininger, MD Baxter Healthcare Corporation
  More Information

No publications provided

Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT01376700     History of Changes
Other Study ID Numbers: 061002, 2011-000410-18
Study First Received: June 17, 2011
Last Updated: April 3, 2013
Health Authority: Austria: Agency for Health and Food Safety
Belarus: Ministry of Health
Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Czech Republic: State Institute for Drug Control
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Paul-Ehrlich-Institut
Greece: National Organization of Medicines
Italy: The Italian Medicines Agency
Lithuania: State Medicine Control Agency - Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Portugal: National Pharmacy and Medicines Institute
Russia: FSI Scientific Center of Expertise of Medical Application
Serbia: Agency for Drugs and Medicinal Devices
Slovakia: State Institute for Drug Control
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Ukraine: State Pharmacological Center - Ministry of Health

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014