Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A - Full Text View

Sponsored by:	Grifols Biologicals Inc.
Information provided by:	Grifols Biologicals Inc.
ClinicalTrials.gov Identifier:	NCT00323856

Purpose

The purpose of this study is to determine the immunologic and overall safety associated with long-term use of Alphanate in subjects diagnosed with severe hemophilia A (Factor VIII:C less than 0.01 IU/ml), who have been previously treated with plasma-derived Factor VIII products other than Alphanate and who have no history of developing either antibody inhibitors to Factor VIII or nonspecific inhibitors of coagulation.

Condition	Intervention	Phase
Severe Hemophilia A	Drug: Alphanate SD	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, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Official Title:	Phase IV A Study of Immunologic Safety for Alphanate in Previously Treated Patients Diagnosed With Severe Hemophilia A

Further study details as provided by Grifols Biologicals Inc.:

Primary Outcome Measures:

Incidence of Factor VIII Inhibitor Development [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Adverse events [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Changes in biochemical parameters indicating renal or hepatic impairment [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Seroconversion to HIV/1, HIV/2, HAV, HBV, HCV or parvovirus B19 in subjects seronegative for these viruses at the time of enrollment [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Amount of product used per year as part of at-home prophylaxis and therapy for bleeding episodes [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Physician's qualitative assessment of hemostasis [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	50
Study Start Date:	January 2003
Estimated Study Completion Date:	December 2010
Estimated Primary Completion Date:	December 2010 (Final data collection date for primary outcome measure)

Intervention Details:

Plasma-derived preparation of Factor VIII

Detailed Description:

This is a Phase IV, non-randomized, multicenter study of at least 50 evaluable subjects diagnosed with severe hemophilia A. Enrolled subjects will be treated at home and with in-clinic therapy exclusively with Alphanate as their sole source of Factor VIII concentrate for prophylaxis and treatment of all bleeding episodes and surgical procedures. Subjects will be treated for at least 2 years and a minimum of 50 exposure days, or if 50 exposure days are not reached, for a maximum of 30 months and in accordance with the subject's usual pre-study treatment regimen. Subjects will continue treatment as above or until they develop inhibitors to Factor VIII at a titer greater than or equal to 5 Bethesda units (BU/ml); Factor VIII becomes ineffective at providing hemostasis, or the subject exhibits severe or serious adverse events that prevent completion of the study.

Eligibility

Ages Eligible for Study:	6 Years to 65 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male
At least 6 years of age and not more than 65 years of age.
Signed and dated Informed Consent Form and Patient Authorization for Release of Information approved by the appropriate Institutional Review Board (IRB) prior to screening and enrollment. If the subject is a minor (i.e., less than 18 years of age) both he and his parent or legal guardian must sign and date the informed consent.
Diagnosis of severe hemophilia A
Levels of Factor VIII less than 0.01 IU/mL.
Treatment with cryoprecipitate, Factor VIII concentrates, and/or whole blood, for at least 150 cumulative exposure days (CEDs) prior to enrollment.
No treatment with cryoprecipitate, Factor VIII concentrate, or any other blood product, for at least 72 hours prior to screening.
No previous diagnosis with inhibitors to Factor VIII at any detectable titer.
Subjects must never have been diagnosed with nonspecific inhibitors of coagulation.
Negative test for the presence of Factor VIII inhibitors at screening and enrollment.
CD4 counts greater than or equal to 400 cells/µL.
Vaccination against hepatitis A and hepatitis B, or evidence of antibodies against hepatitis A and hepatitis B. (A subject who has no prior immunity against hepatitis A will be offered a course of vaccination for hepatitis A.)
Karnofsky Performance Score of at least 50.

Exclusion Criteria:

Any immunosuppressive medications including intravenous immunoglobulins at the time of enrollment.
Clinical signs or symptoms of an infection, such as fever, chills or nausea during screening or enrollment.
History of frequent reactions to Factor VIII concentrates (e.g., chills or headaches).
Prior treatment with Alphanate® (Solvent-Detergent/ Heat-Treated).
Immunocompromised (including HIV+ status or has an impaired immune system due to disease or treatment).

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00323856

Contacts

Contact: Paul Pinciaro, PhD	443-375-8825	paul.pinciaro@grifols.com
Contact: Christine Mackay, PhD	913-681-3078	cmackay@wwcr-cro.com

Locations

United States, District of Columbia
Georgetown University	Recruiting
Washington, District of Columbia, United States, 20007
Contact: Carolyn Francis 202-687-0117 francisc@georgetown.edu
Principal Investigator: Craig Kessler, MD
United States, New York
Mt. Sinai Regional Comprehensive Hemophilia Center	Recruiting
New York, New York, United States, 10029
Contact: Joan McCarthy 212-241-3935 Johanna.McCarthy@msnyuhealth.org
Principal Investigator: Christopher W Walsh, MD
United States, Oklahoma
University of Oklahoma Children's Hospital	Recruiting
Oklahoma City, Oklahoma, United States, 73126
Contact: Felicia Kiplinger 405-271-3661 felicia-kiplinger@ouhsc.edu
Contact: Sarah Hawk, PA 405-271-3661 sarah-hawk@ouhsc.edu
Principal Investigator: Charles Sexauer, MD
United States, Texas
Gulf States Hemophilia and Thrombophilia Center	Recruiting
Houston, Texas, United States, 77030
Contact: Madeline Cantini, BSN, RN, CCRC 713-500-8377 madeline.cantini@uth.tmc.edu
Contact: Kathy Moynihan, RN 713-500-8376 kathrynmoynihan@uth.tmc.edu
Principal Investigator: William K Hoots, MD

Sponsors and Collaborators

Grifols Biologicals Inc.

Investigators

Study Director:

Paul Pinciaro, PhD

Grifols Biologicals Inc.

More Information

Responsible Party:	Grifols Biologicals Inc ( Paul J. Pinciaro, PhD/Director of Clinical Development and Pharmacovigilance )
Study ID Numbers:	GBI 04-01
Study First Received:	May 8, 2006
Last Updated:	March 5, 2008
ClinicalTrials.gov Identifier:	NCT00323856
Health Authority:	United States: Food and Drug Administration

Keywords provided by Grifols Biologicals Inc.:

Hemophilia A
Plasma-derived treatment
Factor VIII
Inhibitor

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
Coagulation Protein Disorders

ClinicalTrials.gov processed this record on January 14, 2009