Clinical trial NCT00157053: 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
Official title: Evaluation of Pharmacokinetics, Safety, Efficacy and Immunogenicity of rAHF-PFM in Previously Treated Hemophilia A Patients - A Continuation Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hemophilia A
Keywords: Factor VIII Deficiency
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2005-09-12
Enrollment: 82
Sex: MALE
Minimum age (years): 10
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 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.


Eligibility criteria: 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

Primary outcomes:
measure: Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)
timeFrame: 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 outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Mountain States Regional Hemophilia and Thrombosis Center
state: Colorado
city: Atlanta
country: United States
facility: Childrens Healthcare of Atlanta Blood Bank
state: Georgia
city: Peoria
country: United States
facility: Comprehensive Bleeding Disorders Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia & Thrombosis Center
state: Indiana
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: New Brunswick
country: United States
facility: University of Medicine & Dentistry of New Jersey
state: New Jersey
city: New York
country: United States
facility: Cornell Medical Center
state: New York
city: New York
country: United States
facility: Mt. Sinai Medical School
state: New York
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center Pharmacy
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
city: Vienna
country: Austria
facility: Allgemeines Krankenhaus der Stadt Wien
city: Leuven
country: Belgium
facility: KU Leuven Universitaire Ziekenhuizen
city: Calgary
country: Canada
facility: University of Alberta
state: Alberta
city: Lyon
country: France
facility: Hôpital Edouard Herriot
city: Frankfurt am Main
country: Germany
facility: Klinikum der J. W. Goethe - Universität
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Munich
country: Germany
facility: Klinikum Innenstadt der Universität München
city: Milan
country: Italy
facility: Ospedale Maggiore di Milano
city: Malmö
country: Sweden
facility: University Hospital MAS
city: London
country: United Kingdom
facility: The Royal Free Hospital
state: England
city: Manchester
country: United Kingdom
facility: Central Manchester Healthcare NHS Trust
state: England
city: Oxford
country: United Kingdom
facility: The Churchill Hospital
state: England
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
state: Wales
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.