Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery

Phase 2

Completed

• Conditions: Hemophilia A

• Registration Number: NCT00157105
• Lead Sponsor: Baxalta now part of Shire

Brief Summary

The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery.

Detailed Description

Not available

Study Timeline

• Study Start: 2001-02-12
• Primary Completion: 2004-08-03
• Study Completion: 2004-08-03
• Study First Submitted: 2005-09-08
• Study First Submitted QC: 2005-09-08
• Study First Posted: 2005-09-12
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-22
• Last Update Posted: 2021-08-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 59

Inclusion Criteria

• Age >= 5 years
• The subject has severe or moderately severe hemophilia A defined by a baseline factor VIII level <= 2% of normal documented at screening or historically (e.g., at hemophilia diagnosis)
• Subjects may enroll regardless of their serologic status for human immunodeficiency virus (HIV-1) and hepatitis C virus (HCV)
• The subject requires a surgical, dental or other invasive procedure--either elective or emergency
• The subject has a history of at least 150 exposure days for all other factor VIII products (as estimated by the study site investigator) prior to study entry
• The subject has a life expectancy of at least 28 days from the day of surgery
• The subject has been informed of the nature of the study, agreed to its provisions, and signed and dated the informed consent form approved by the appropriate IRB/IEC and Baxter BioScience

Exclusion Criteria

• The subject has a detectable inhibitor to factor VIII in the local hemostasis laboratory at the investigative site at the time of enrollment
• The subject has a history of inhibitor to factor VIII > 1.0 BU. Note: If the subject has a history of an inhibitor titer > 1.0 BU at any time prior to enrollment but demonstrated expected clinical responses to conventional doses of factor VIII therapy, the subject may enroll
• The subject has known hypersensitivity to Recombinate
• The subject is currently participating in another investigational drug study, or has participated in any clinical trial involving an investigational drug within 30 days of study entry
• The subject has clinical and/or laboratory evidence of abnormal hemostasis from causes other than hemophilia A (e.g., late-stage chronic liver disease, immune thrombocytopenic purpura, disseminated intravascular coagulation)
• The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Percent of participants experiencing treatment-related adverse experiences (AEs)	Throughout the study period of approximately 3.5 years

Trial Locations

Locations (10)

Michigan State University; 🇺🇸 East Lansing, Michigan, United States

Hemophilia Center of Western Pennsylvania; 🇺🇸 Pittsburgh, Pennsylvania, United States

Mt. Sinai Medical School, Hemophilia Comprehensive Care Center; 🇺🇸 New York, New York, United States

Children´s Hospital Medical Center Pharmacy, Hemophilia Treatment Center; 🇺🇸 Cincinnati, Ohio, United States

Children´s Hospital Los Angeles, Hemophilia Comprehensive Care Center, Division of Pediatric Hematology/Oncology; 🇺🇸 Los Angeles, California, United States

Children´s Healthcare of Atlanta Blood Bank; 🇺🇸 Atlanta, Georgia, United States

Indiana Hemophilia & Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States

University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics; 🇺🇸 Iowa City, Iowa, United States

Brigham and Women´s Hospital, Hematology Division; 🇺🇸 Boston, Massachusetts, United States

Puget Sound Blood Center; 🇺🇸 Seattle, Washington, United States