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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00323856
Recruitment Status : Active, not recruiting
First Posted : May 10, 2006
Last Update Posted : February 5, 2020
Information provided by (Responsible Party):
Grifols Biologicals, LLC

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.

Condition or disease Intervention/treatment Phase
Severe Hemophilia A Drug: Alphanate SD/HT Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase IV A Study of Immunologic Safety for Alphanate in Previously Treated Patients Diagnosed With Severe Hemophilia A
Actual Study Start Date : January 2003
Actual Primary Completion Date : December 2018
Estimated Study Completion Date : July 2020

Arm Intervention/treatment
Experimental: Coagulation factor VIII (Human)
Anti-Hemophilic coagulation factor VIII (Human) Alphanate SD/HT
Drug: Alphanate SD/HT
Plasma-derived preparation of Factor VIII
Other Name: Anti-hemophilic (human) coagulation factor VIII

Primary Outcome Measures :
  1. Incidence of Factor VIII Inhibitor Development [ Time Frame: 24 months ]

Secondary Outcome Measures :
  1. Adverse events [ Time Frame: 24 months ]
  2. Changes in biochemical parameters indicating renal or hepatic impairment [ Time Frame: 24 months ]
  3. 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 ]
  4. Amount of product used per year as part of at-home prophylaxis and therapy for bleeding episodes [ Time Frame: 24 months ]
  5. Physician's qualitative assessment of hemostasis [ Time Frame: 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00323856

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Oddzial Chorob Wewnetrznych i Hematologii
Poznan, Szkolna, Poland
Katedra i Klinika Hematologii Collegium Medicum UJ
Krakow, Poland
Sponsors and Collaborators
Grifols Biologicals, LLC
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Study Director: Paul J Pinciaro, PhD Grifols Biologicals, LLC
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Responsible Party: Grifols Biologicals, LLC Identifier: NCT00323856    
Other Study ID Numbers: GBI 04-01
First Posted: May 10, 2006    Key Record Dates
Last Update Posted: February 5, 2020
Last Verified: February 2020
Keywords provided by Grifols Biologicals, LLC:
Hemophilia A
Plasma-derived treatment
Factor VIII
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII