Phase 4
N=50
Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
Severe Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT00323856 ↗Enrolled (actual)
50
Serious AEs
8.0%
Results posted
Jun 2022
Primary outcome: Primary: Number of Participants With Factor VIII (FVIII) Inhibitor Development — 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Alphanate SD/HT (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- Male
- Sponsor
- Grifols Biologicals, LLC
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Factor VIII (FVIII) Inhibitor Development |
— | — |
| SECONDARY Number of Participants With Adverse Events (AE) |
30 | — |
| SECONDARY Change From Baseline in Alkaline Phosphatase |
2.315; -0.030; -0.146; -0.087; -0.309; -0.127 | — |
| SECONDARY Change From Baseline in Alanine Aminotransferase |
0.577; -0.005; -0.055; -0.089; -0.114; -0.081 | — |
| SECONDARY Change From Baseline in Aspartate Aminotransferase |
0.544; 0.023; -0.011; -0.062; -0.092; -0.071 | — |
| SECONDARY Change From Baseline in Lactate Dehydrogenase |
5.64; -0.07; -0.04; -0.01; -0.31; -0.47 | — |
| SECONDARY Change From Baseline in Bilirubin |
12.346; 41.387; -0.815; 40.485; 1.461; -0.031 | — |
| SECONDARY Change From Baseline in Blood Urea Nitrogen |
5.330; 0.140; 0.177; 0.489; 1.356; 13.476 | — |
| SECONDARY Change From Baseline in Creatinine |
61.30; 3.47; -1.76; 4.18; 18.56; 5.80 | — |
| 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 |
5; 21; 0; 6; 3; 6 | — |
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.
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).
Data sourced from ClinicalTrials.gov (NCT00323856). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.