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Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901

Phase 2
Completed
Conditions
Hemophilia A
Registration Number
NCT00157053
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 subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation of efficacy and safety. The study is open to patients who completed Baxter Study 069901.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
82
Inclusion Criteria
  • Subject has completed Baxter protocol 069901
  • Subject is HIV-1 seronegative or, if HIV-1 seropositive, has a CD4+ lymphocyte count >= 400/mm3 documented within three months of the screening visit
  • Subject (and his legally acceptable representative, in the case of study participants >= 10 and < 18 years of age) 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
Exclusion Criteria
  • The subject received factor VIII products other than rAHF-PFM upon completion of Baxter protocol 069901
  • The subject developed an inhibitor to factor VIII, measured in the central laboratory, during Baxter protocol 069901. An inhibitor is defined as a Bethesda titer > 1.0 or, if Bethesda titer < 1.0, confirmation using the Nijmegen modification of the Bethesda assay with a titer > 0.6
  • The subject is scheduled to receive an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids) during the course of the study
  • 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
NameTimeMethod
Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion.
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (24)

Medizinische Hochschule Hannover

🇩🇪

Hannover, Germany

Allgemeines Krankenhaus der Stadt Wien

🇦🇹

Vienna, Austria

University of Alberta

🇨🇦

Calgary, Alberta, Canada

KU Leuven Universitaire Ziekenhuizen

🇧🇪

Leuven, Belgium

Klinikum der J. W. Goethe - Universität

🇩🇪

Frankfurt am Main, Germany

Hôpital Edouard Herriot

🇫🇷

Lyon, France

Central Manchester Healthcare NHS Trust

🇬🇧

Manchester, England, United Kingdom

Indiana Hemophilia & Thrombosis Center

🇺🇸

Indianapolis, Indiana, United States

The Churchill Hospital

🇬🇧

Oxford, England, United Kingdom

University Hospital of Wales

🇬🇧

Cardiff, Wales, United Kingdom

Puget Sound Blood Center

🇺🇸

Seattle, Washington, United States

The Royal Free Hospital

🇬🇧

London, England, United Kingdom

Klinikum Innenstadt der Universität München

🇩🇪

Munich, Germany

Ospedale Maggiore di Milano

🇮🇹

Milan, Italy

Childrens Healthcare of Atlanta Blood Bank

🇺🇸

Atlanta, Georgia, United States

University Hospital MAS

🇸🇪

Malmö, Sweden

Michigan State University

🇺🇸

East Lansing, Michigan, United States

Mountain States Regional Hemophilia and Thrombosis Center

🇺🇸

Aurora, Colorado, United States

Comprehensive Bleeding Disorders Center

🇺🇸

Peoria, Illinois, United States

Cornell Medical Center

🇺🇸

New York, New York, United States

Children's Hospital Medical Center Pharmacy

🇺🇸

Cincinnati, Ohio, United States

University of Medicine & Dentistry of New Jersey

🇺🇸

New Brunswick, New Jersey, United States

Mt. Sinai Medical School

🇺🇸

New York, New York, United States

Hemophilia Center of Western Pennsylvania

🇺🇸

Pittsburgh, Pennsylvania, United States

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