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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

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Antihemophilic factor, recombinant, manufactured protein-free
Sponsored by
Baxalta now part of Shire
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Factor VIII Deficiency

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Mountain States Regional Hemophilia and Thrombosis Center
  • Childrens Healthcare of Atlanta Blood Bank
  • Comprehensive Bleeding Disorders Center
  • Indiana Hemophilia & Thrombosis Center
  • Michigan State University
  • University of Medicine & Dentistry of New Jersey
  • Cornell Medical Center
  • Mt. Sinai Medical School
  • Children's Hospital Medical Center Pharmacy
  • Hemophilia Center of Western Pennsylvania
  • Puget Sound Blood Center
  • Allgemeines Krankenhaus der Stadt Wien
  • KU Leuven Universitaire Ziekenhuizen
  • University of Alberta
  • Hôpital Edouard Herriot
  • Klinikum der J. W. Goethe - Universität
  • Medizinische Hochschule Hannover
  • Klinikum Innenstadt der Universität München
  • Ospedale Maggiore di Milano
  • University Hospital MAS
  • The Royal Free Hospital
  • Central Manchester Healthcare NHS Trust
  • The Churchill Hospital
  • University Hospital of Wales

Outcomes

Primary Outcome Measures

Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)

Secondary Outcome Measures

Full Information

First Posted
September 8, 2005
Last Updated
August 22, 2021
Sponsor
Baxalta now part of Shire
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1. Study Identification

Unique Protocol Identification Number
NCT00157053
Brief Title
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

2. Study Status

Record Verification Date
August 2021
Overall Recruitment Status
Completed
Study Start Date
November 22, 2001 (Actual)
Primary Completion Date
August 3, 2004 (Actual)
Study Completion Date
August 3, 2004 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baxalta now part of Shire

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Factor VIII Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
82 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Antihemophilic factor, recombinant, manufactured protein-free
Primary Outcome Measure Information:
Title
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.

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Mountain States Regional Hemophilia and Thrombosis Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Childrens Healthcare of Atlanta Blood Bank
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Comprehensive Bleeding Disorders Center
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61614
Country
United States
Facility Name
Indiana Hemophilia & Thrombosis Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Michigan State University
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48824
Country
United States
Facility Name
University of Medicine & Dentistry of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08903-0019
Country
United States
Facility Name
Cornell Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Mt. Sinai Medical School
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Children's Hospital Medical Center Pharmacy
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Hemophilia Center of Western Pennsylvania
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213-4306
Country
United States
Facility Name
Puget Sound Blood Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104-1256
Country
United States
Facility Name
Allgemeines Krankenhaus der Stadt Wien
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
KU Leuven Universitaire Ziekenhuizen
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University of Alberta
City
Calgary
State/Province
Alberta
ZIP/Postal Code
470 MSB
Country
Canada
Facility Name
Hôpital Edouard Herriot
City
Lyon
ZIP/Postal Code
69437
Country
France
Facility Name
Klinikum der J. W. Goethe - Universität
City
Frankfurt am Main
ZIP/Postal Code
60590
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
30623
Country
Germany
Facility Name
Klinikum Innenstadt der Universität München
City
Munich
ZIP/Postal Code
D-80336
Country
Germany
Facility Name
Ospedale Maggiore di Milano
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
University Hospital MAS
City
Malmö
ZIP/Postal Code
S-20502
Country
Sweden
Facility Name
The Royal Free Hospital
City
London
State/Province
England
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Central Manchester Healthcare NHS Trust
City
Manchester
State/Province
England
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
The Churchill Hospital
City
Oxford
State/Province
England
ZIP/Postal Code
OX3 7LJ
Country
United Kingdom
Facility Name
University Hospital of Wales
City
Cardiff
State/Province
Wales
ZIP/Postal Code
CF4 4XW
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
19216617
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.
Results Reference
derived

Learn more about this trial

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

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