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

August 22, 2021 updated by: Baxalta now part of Shire

Evaluation of Pharmacokinetics, Safety, Efficacy and Immunogenicity of rAHF-PFM in Previously Treated Hemophilia A Patients - A Continuation Study

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.

Study Overview

Status

Completed

Conditions

Hemophilia A

Intervention / Treatment

Drug: Antihemophilic factor, recombinant, manufactured protein-free

Study Type

Interventional

Enrollment (Actual)

Phase

Phase 2
Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Austria
- - Vienna, Austria, 1090
    - Allgemeines Krankenhaus der Stadt Wien
Belgium
- - Leuven, Belgium, 3000
    - KU Leuven Universitaire Ziekenhuizen
Canada
- Alberta
  - Calgary, Alberta, Canada, 470 MSB
    - University of Alberta
France
- - Lyon, France, 69437
    - Hôpital Edouard Herriot
Germany
- - Frankfurt am Main, Germany, 60590
    - Klinikum der J. W. Goethe - Universität
  - Hannover, Germany, 30623
    - Medizinische Hochschule Hannover
  - Munich, Germany, D-80336
    - Klinikum Innenstadt der Universität München
Italy
- - Milan, Italy, 20122
    - Ospedale Maggiore di Milano
Sweden
- - Malmö, Sweden, S-20502
    - University Hospital MAS
United Kingdom
- England
  - London, England, United Kingdom, NW3 2QG
    - The Royal Free Hospital
  - Manchester, England, United Kingdom, M13 9WL
    - Central Manchester Healthcare NHS Trust
  - Oxford, England, United Kingdom, OX3 7LJ
    - The Churchill Hospital
- Wales
  - Cardiff, Wales, United Kingdom, CF4 4XW
    - University Hospital of Wales
United States
- Colorado
  - Aurora, Colorado, United States, 80045
    - Mountain States Regional Hemophilia and Thrombosis Center
- Georgia
  - Atlanta, Georgia, United States, 30322
    - Childrens Healthcare of Atlanta Blood Bank
- Illinois
  - Peoria, Illinois, United States, 61614
    - Comprehensive Bleeding Disorders Center
- Indiana
  - Indianapolis, Indiana, United States, 46260
    - Indiana Hemophilia & Thrombosis center
- Michigan
  - East Lansing, Michigan, United States, 48824
    - Michigan State University
- New Jersey
  - New Brunswick, New Jersey, United States, 08903-0019
    - University Of Medicine & Dentistry Of New Jersey
- New York
  - New York, New York, United States, 10021
    - Cornell Medical Center
  - New York, New York, United States, 10029
    - Mt. Sinai Medical School
- Ohio
  - Cincinnati, Ohio, United States, 45229
    - Children's Hospital Medical Center Pharmacy
- Pennsylvania
  - Pittsburgh, Pennsylvania, United States, 15213-4306
    - Hemophilia Center of Western Pennsylvania
- Washington
  - Seattle, Washington, United States, 98104-1256
    - Puget Sound Blood Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

Genders Eligible for Study

Male

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Interventional Model: Single Group Assignment
Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM) Time Frame: 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.	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.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Baxalta now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 22, 2001

Primary Completion (Actual)

August 3, 2004

Study Completion (Actual)

August 3, 2004

Study Registration Dates

First Submitted

September 8, 2005

First Submitted That Met QC Criteria