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

NCT00157105 | Completed Phase 2

• 1 other identifier: 069902
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 10

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Percent of participants experiencing treatment-related adverse experiences (AEs)

  Throughout the study period of approximately 3.5 years

Interventions

Antihemophilic factor, recombinant, manufactured protein-free DRUG

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (10)

Children´s Hospital Los Angeles, Hemophilia Comprehensive Care Center, Division of Pediatric Hematology/Oncology

Los Angeles, California, 90027, United States

Location

Children´s Healthcare of Atlanta Blood Bank

Atlanta, Georgia, 30322, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics

Iowa City, Iowa, 52242, United States

Location

Brigham and Women´s Hospital, Hematology Division

Boston, Massachusetts, 02115, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Mt. Sinai Medical School, Hemophilia Comprehensive Care Center

New York, New York, 10029, United States

Location

Children´s Hospital Medical Center Pharmacy, Hemophilia Treatment Center

Cincinnati, Ohio, 45229, United States

Location

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213-4306, United States

Location

Puget Sound Blood Center

Seattle, Washington, 98104-1256, United States

Location

Related Publications (1)

• 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.

  PMID: 19216617 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2005
• First Posted: September 12, 2005
• Study Start: February 12, 2001
• Primary Completion: August 3, 2004
• Study Completion: August 3, 2004
• Last Updated: August 24, 2021

Record last verified: 2021-08

Locations

US (10)