Have you or your loved ones been diagnosed with severe hemophilia a?
You may be eligible to participate in a severe hemophilia a clinical trial.
Have you or your loved ones been diagnosed with severe hemophilia a? You may be eligible to participate in a severe hemophilia a clinical trial.
What is a clinical trial? Is participating in a clinical trial right for you? Learn more
Severe Hemophilia A Clinical Trial in Krakow
Have you or your loved ones been diagnosed with severe hemophilia a?
You may be eligible to participate in a severe hemophilia a clinical trial.
Have you or your loved ones been diagnosed with severe hemophilia a? You may be eligible to participate in a severe hemophilia a clinical trial.
Completed
Male
6 - 65
Years old
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.
Details for the study
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 <br /> long-term use of Alphanate in subjects diagnosed with severe hemophilia A (Factor VIII:C less <br /> than 0.01 IU/ml), who have been previously treated with plasma-derived Factor VIII products <br /> other than Alphanate and who have no history of developing either antibody inhibitors to <br /> 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.
Treatments and/or Procedures
Alphanate SD HT
Plasma-derived preparation of Factor VIII
Outcome Measures
Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.
Primary
Number of Participants With Factor VIII (FVIII) Inhibitor Development
Incidence of FVIII inhibitor development was defined as any result determined positive at a central laboratory (inhibitor titer of greater than 0.6 modified Bethesda Units/milliliters [BU/mL]) using Nijmegen modification of the Bethesda assay.
Secondary
Change From Baseline in Alanine Aminotransferase
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Number of Participants Human Immunodeficiency Virus Type 1 and 2 (HIV-1/HIV-2), Hepatitis A Virus (HAV), Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or Parvovirus B19 -Negative at Baseline Who Are Seropositive for Any of These Viruses
Seroconversion based on Enzyme-linked Immunosorbent Assay (ELISA). Seronegative defined as non-reactive in an ELISA test for antibody to the virus in question. Seropositive defined as reactive in an ELISA test for antibody to the virus in question.
Secondary
Change From Baseline in Creatinine
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Change From Baseline in Blood Urea Nitrogen
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Change From Baseline in Bilirubin
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Change From Baseline in Aspartate Aminotransferase
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Change From Baseline in Alkaline Phosphatase
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Secondary
Number of Participants With Adverse Events (AE)
An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant administered a medicinal product or study treatment, and which did not necessarily have a causal relationship with this administration. Here end of study is defined as completion/discontinuation visit.
Secondary
Change From Baseline in Lactate Dehydrogenase
The Baseline value was the last non-missing value before study drug was taken and end of study was defined as completion/discontinuation visit. Change from Baseline was calculated by subtracting Baseline value from the post-infusion visit value.
Study 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).