Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A - Full Text View

Sponsored by:	Baxter Healthcare Corporation
Information provided by:	Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:	NCT00243386

Purpose

The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40 IU/kg every 48 ± 6 hours; actual dose determined by the investigator) with that of alternate prophylaxis (20-80 IU/kg every 72 + 6 hours; actual dose determined by Baxter utilizing an algorithm and the patient's pharmacokinetic data). The rates of bleeding episodes for the on-demand regimen and the prophylaxis regimens will also be compared. Enrolled patients will be treated originally on demand for a period of 6 months and then they will be randomized into one of the prophylaxis arms. Prophylactic treatment will last for a period of 12 months +/- 2 weeks.

Condition	Intervention	Phase
Hemophilia A	Drug: Antihemophilic factor, recombinant, manufactured protein-free	Phase IV

Genetics Home Reference related topics: hemophilia

MedlinePlus related topics: Hemophilia

Drug Information available for: Factor VIII Octocog alfa

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Efficacy Study
Official Title:	Advate Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (ADVATE rAHF-PFM): A Phase 4 Study Comparing Two Prophylactic Regimens in Subjects With Severe or Moderately Severe Hemophilia A

Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:

Yearly transformed rate of bleeding episodes estimated from each arm of the 1 year prophylactic regimen [ Time Frame: Yearly ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Yearly transformed rate of bleeding episodes estimated from a six-month on demand treatment regimen and each arm of the 1 year prophylactic regimen [ Time Frame: Yearly ] [ Designated as safety issue: No ]
Subjects who develop a factor VIII inhibitor (and 95% confidence intervals for risk of inhibitor development) [ Time Frame: No time frame ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	66
Study Start Date:	February 2006
Estimated Primary Completion Date:	December 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Active Comparator Standard prophylaxis	Drug: Antihemophilic factor, recombinant, manufactured protein-free Standard prophylaxis: 20-40 IU/kg every 48+/-6 hours, actual dose determined by investigator
2: Experimental Alternate prophylaxis	Drug: Antihemophilic factor, recombinant, manufactured protein-free Alternate prophylaxis: 20-80 IU/kg every 72+/-6 hours, actual dose determined by Baxter using an algorithm and the patient´s pharmacokinetic data

Eligibility

Ages Eligible for Study:	7 Years to 65 Years
Genders Eligible for Study:	Male
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 tested at screening
The subject has a documented history of at least 150 exposure days to factor VIII concentrates (either plasma-derived or recombinant)
The subject is within 7 to 65 years of age
The subject has a Karnofsky performance score > (greater than) 60
The subject is human immunodeficiency virus negative (HIV-) or is HIV+ with a CD4 count >= 400 cells/mm³ (CD4 count determined at screening, if necessary)
The subject has been on a documented on-demand treatment regimen for at least 12 months immediately prior to enrollment
The subject has a documented history (e.g. in medical charts or dispensing information) of at least 8 joint hemorrhages in the 12 months immediately prior to enrollment
The subject resides within the coverage area of the mobile compliance device; coverage area will be determined at screening
The subject or the subject's legally authorized representative has provided written informed consent

Exclusion Criteria:

The subject has a known hypersensitivity to factor VIII concentrates or mouse or hamster proteins
The subject has a history of factor VIII inhibitors with a titer >= 0.6 BU (by Bethesda or Nijmegen assay) at any time prior to screening
The subject has a detectable factor VIII inhibitor at screening, with a titer >= 0.4 BU (by Nijmegen Assay) in the central laboratory
The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand's Disease)
The subject has been treated during the last sixty (60) days prior to or is being treated at screening/enrollment with an immunomodulating drug.
The subject has participated in another investigational study within 30 days of enrollment
The subject has previously participated in a clinical study with rAHF-PFM
The subject's clinical condition may require a moderate or major surgery (defined as intra-operative estimated blood loss greater than 500 mL) during the period of the subject's participation in the study

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00243386

Show 22 Study Locations

Sponsors and Collaborators

Baxter Healthcare Corporation

Investigators

Principal Investigator:

Peter Collins, MD

Cardiff and Vale NHS

More Information

Responsible Party:	Baxter Healthcare Corporation ( Minal Ashtekar, Clinical Project Manager (US); Christiane Thomasser, Clinical Project Manager (EU) )
Study ID Numbers:	060201
Study First Received:	October 21, 2005
Last Updated:	February 4, 2008
ClinicalTrials.gov Identifier:	NCT00243386
Health Authority:	United States: Food and Drug Administration; Austria: Federal Ministry for Health and Women; Czech Republic: State Institute for Drug Control; Greece: Ministry of Health and Welfare; Hungary: National Institute of Pharmacy; Italy: Ministry of Health; Slovenia: Agency for Medicinal Products - Ministry of Health; Spain: Spanish Agency of Medicines; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Study placed in the following topic categories:

Hemorrhagic Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Blood Coagulation Disorders

Hemophilia A
Hemostatic Disorders
Factor VIII

Additional relevant MeSH terms:

Blood Coagulation Disorders, Inherited
Coagulants
Coagulation Protein Disorders

Therapeutic Uses
Hematologic Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 13, 2009