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

NCT00157053 | Completed Phase 2

• 1 other identifier: 060102
• Study Type: interventional
• Target: 82
• Locations: 9 countries
• Sites: 24

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.

Conditions

Hemophilia A

Keywords

Factor VIII Deficiency

Outcome Measures

Primary Outcomes (1)

• 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.

Interventions

Antihemophilic factor, recombinant, manufactured protein-free DRUG

Eligibility Criteria

• Age: 10 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (24)

Mountain States Regional Hemophilia and Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Childrens Healthcare of Atlanta Blood Bank

Atlanta, Georgia, 30322, United States

Location

Comprehensive Bleeding Disorders Center

Peoria, Illinois, 61614, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

University of Medicine & Dentistry of New Jersey

New Brunswick, New Jersey, 08903-0019, United States

Location

Cornell Medical Center

New York, New York, 10021, United States

Location

Mt. Sinai Medical School

New York, New York, 10029, United States

Location

Children's Hospital Medical Center Pharmacy

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

Allgemeines Krankenhaus der Stadt Wien

Vienna, 1090, Austria

Location

KU Leuven Universitaire Ziekenhuizen

Leuven, 3000, Belgium

Location

University of Alberta

Calgary, Alberta, 470 MSB, Canada

Location

Hôpital Edouard Herriot

Lyon, 69437, France

Location

Klinikum der J. W. Goethe - Universität

Frankfurt am Main, 60590, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30623, Germany

Location

Klinikum Innenstadt der Universität München

Munich, D-80336, Germany

Location

Ospedale Maggiore di Milano

Milan, 20122, Italy

Location

University Hospital MAS

Malmo, S-20502, Sweden

Location

The Royal Free Hospital

London, England, NW3 2QG, United Kingdom

Location

Central Manchester Healthcare NHS Trust

Manchester, England, M13 9WL, United Kingdom

Location

The Churchill Hospital

Oxford, England, OX3 7LJ, United Kingdom

Location

University Hospital of Wales

Cardiff, Wales, CF4 4XW, United Kingdom

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: November 22, 2001
• Primary Completion: August 3, 2004
• Study Completion: August 3, 2004
• Last Updated: August 24, 2021

Record last verified: 2021-08

Locations

AU (1); DE (3); US (11); IT (1); BE (1); CA (1); GB (4); SE (1); FR (1)