Clinical trial NCT00157105: Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery
Official title: Recombinant Antihemophilic Factor (rAHF PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Safety and Efficacy in Hemophilia A Patients Undergoing Surgical or Invasive Procedures
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2005-09-12
Enrollment: 59
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Percent of participants experiencing treatment-related adverse experiences (AEs)
timeFrame: Throughout the study period of approximately 3.5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children´s Hospital Los Angeles, Hemophilia Comprehensive Care Center, Division of Pediatric Hematology/Oncology
state: California
city: Atlanta
country: United States
facility: Children´s Healthcare of Atlanta Blood Bank
state: Georgia
city: Indianapolis
country: United States
facility: Indiana Hemophilia & Thrombosis Center
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics
state: Iowa
city: Boston
country: United States
facility: Brigham and Women´s Hospital, Hematology Division
state: Massachusetts
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: New York
country: United States
facility: Mt. Sinai Medical School, Hemophilia Comprehensive Care Center
state: New York
city: Cincinnati
country: United States
facility: Children´s Hospital Medical Center Pharmacy, Hemophilia Treatment Center
state: Ohio
city: Pittsburgh
country: United States
facility: Hemophilia Center of Western Pennsylvania
state: Pennsylvania
city: Seattle
country: United States
facility: Puget Sound Blood Center
state: Washington
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther. 2009 Mar;9(3):273-83. doi: 10.1517/14712590902729392.