Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients - Full Text View

Sponsor:	Baxter Healthcare Corporation
Information provided by:	Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:	NCT00157157

Purpose

The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII before.

Condition	Intervention	Phase
Hemophilia A	Drug: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM)	Phase III

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study
Official Title:	Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (rAHF-PFM): Evaluation of Immunogenicity, Efficacy, and Safety in Previously Untreated Patients With Hemophilia A

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia Help Me Understand Genetics

MedlinePlus related topics: Hemophilia Surgery

Drug Information available for: Octocog alfa Moroctocog Alfa Factor VIII

U.S. FDA Resources

Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:

To assess the immunogenicity of rAHF PFM by determining the risk of factor VIII inhibitor development [ Time Frame: Inhibitor assessments at pre-determined intervals ] [ Designated as safety issue: Yes ]
To assess in vivo incremental recovery of rAHF PFM [ Time Frame: At the first study visit, at every other interval study visit, and at the study termination visit ] [ Designated as safety issue: No ]
To evaluate the hemostatic efficacy of rAHF PFM in the management of acute bleeding events [ Time Frame: No time frame ] [ Designated as safety issue: No ]
To evaluate the hemostatic efficacy of rAHF PFM during surgical prophylaxis, if surgery is required [ Time Frame: No time frame ] [ Designated as safety issue: No ]
To assess the safety of rAHF-PFM [ Time Frame: Throughout the study ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	50
Study Start Date:	April 2004
Study Completion Date:	September 2009
Primary Completion Date:	September 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Active Comparator Prophylactic treatment	Drug: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) Prophylactic regimen of 25-50 IU/kg 3-4 times per week; surgical prophylaxis and episodic treatments of new bleeding episodes are also possible (treatment regimen at discretion of investigator). Study product (rAHF PFM) will be administered intravenously over a period of <= 5 minutes at a maximum infusion rate of 10 mL/minute.
2: Active Comparator Modified prophylactic treatment	Drug: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) Modified prophylactic treatment (in pre-determined fashion) to prevent bleeding; surgical prophylaxis and episodic treatments of new bleeding episodes are also possible (treatment regimen at discretion of investigator). Study product (rAHF PFM) will be administered intravenously over a period of <= 5 minutes at a maximum infusion rate of 10 mL/minute.
3: Active Comparator On-demand treatment	Drug: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) On-demand treatment (as determined by the investigator); surgical prophylaxis and episodic treatments of new bleeding episodes are also possible (treatment regimen at discretion of investigator). Study product (rAHF PFM) will be administered intravenously over a period of <= 5 minutes at a maximum infusion rate of 10 mL/minute.

Eligibility

Ages Eligible for Study:	up to 6 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level <= 2% of normal, as documented at screening
The subject is < 6 years of age
The subject's legally authorized representative has provided written informed consent

Exclusion Criteria:

The subject has a history of exposure to factor VIII other than rAHF PFM or more than 3 infusions of commercially available rAHF PFM (i.e., ADVATE) within 28 days prior to screening, as determined by the subject's medical history. Any infusion of factor VIII replacement products prior to the 28-day period excludes the subject from participation
The subject has received more than 3 infusions of rAHF PFM (commercially available and/or study product) between screening and prior to the initial recovery infusion
The subject has a detectable inhibitor to factor VIII, as measured in the screening sample by the Nijmegen assay in the central laboratory
The subject has a history of inhibitor to factor VIII at any time prior to screening
The subject has a known hypersensitivity to rAHF PFM
The subject has any 1 of the following laboratory abnormalities at the time of screening:
1. Platelet count < 100,000/mm3
2. Hemoglobin concentration < 10 g/dL (100 g/L)
3. Serum creatinine > 1.5 times the upper limit of normal (ULN) for age
4. Total bilirubin > 2 times the ULN for age
The subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand's disease)
The subject is known to be seropositive for human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV), as determined by the subject's medical history
At the time of enrollment, the subject has a clinically significant chronic disease other than hemophilia A
The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 120 days of the screening visit
The subject (or the subject's legally authorized representative) is identified by the investigator as being unable or unwilling to cooperate with study procedures
The subject has received any blood product, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00157157

Show 36 Study Locations

Sponsors and Collaborators

Baxter Healthcare Corporation

Investigators

Principal Investigator:

Baxter BioScience Investigator

Baxter BioScience

More Information

No publications provided by Baxter Healthcare Corporation

Additional publications automatically indexed to this study by National Clinical Trials Identifier (NCT ID):

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.

Responsible Party:	Baxter Healthcare Corporation ( Julie McLaren, Clinical Project Manager (US); Michael Zoerer, Clinical Project Manager (EU) )
Study ID Numbers:	060103
Study First Received:	September 9, 2005
Last Updated:	September 16, 2009
ClinicalTrials.gov Identifier:	NCT00157157 History of Changes
Health Authority:	United States: Food and Drug Administration; Germany: Paul-Ehrlich-Institut; Austria: Federal Ministry for Health and Women; France: Afssaps - French Health Products Safety Agency; Italy: Ministry of Health; Spain: Spanish Agency of Medicines; Sweden: Medical Products Agency; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Baxter Healthcare Corporation:

Factor VIII Deficiency

Additional relevant MeSH terms:

Hemorrhagic Disorders
Blood Coagulation Disorders, Inherited
Coagulants
Genetic Diseases, Inborn
Coagulation Protein Disorders
Hematologic Diseases

Therapeutic Uses
Blood Coagulation Disorders
Hematologic Agents
Hemophilia A
Pharmacologic Actions
Factor VIII

ClinicalTrials.gov processed this record on February 08, 2010