Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A

• Status: Active, not recruiting
• Trial end date: 2020-07-01
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Grifols Biologicals Inc.; Grifols Biologicals, LLC
• Treatments: Factor VIII

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).