Brief Title
Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
Official Title
Phase IV A Study of Immunologic Safety for Alphanate in Previously Treated Patients Diagnosed With Severe Hemophilia A
Brief 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.
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.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Number of Participants With Factor VIII (FVIII) Inhibitor Development
Secondary Outcome
Number of Participants With Adverse Events (AE)
Condition
Severe Hemophilia A
Intervention
Alphanate SD/HT
Study Arms / Comparison Groups
Coagulation factor VIII (Human)
Description: Anti-Hemophilic coagulation factor VIII (Human) Alphanate SD/HT
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
51
Start Date
April 8, 2003
Completion Date
December 14, 2018
Primary Completion Date
December 11, 2018
Eligibility 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).
Gender
Male
Ages
6 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Michael Ken Woodward, ,
Location Countries
Poland
Location Countries
Poland
Administrative Informations
NCT ID
NCT00323856
Organization ID
GBI 04-01
Responsible Party
Sponsor
Study Sponsor
Grifols Biologicals, LLC
Study Sponsor
Michael Ken Woodward, Study Director, Instituto Grifols SA
Verification Date
March 2022