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

This study is currently recruiting participants.
Verified February 2014 by Grifols Biologicals Inc.
Sponsor:
Information provided by (Responsible Party):
Grifols Biologicals Inc.
ClinicalTrials.gov Identifier:
NCT00323856
First received: May 8, 2006
Last updated: February 17, 2014
Last verified: February 2014

May 8, 2006
February 17, 2014
January 2003
December 2019   (final data collection date for primary outcome measure)
Incidence of Factor VIII Inhibitor Development [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Incidence of Factor VIII Inhibitor Development
Complete list of historical versions of study NCT00323856 on ClinicalTrials.gov Archive Site
  • Adverse events [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
  • Changes in biochemical parameters indicating renal or hepatic impairment [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • Amount of product used per year as part of at-home prophylaxis and therapy for bleeding episodes [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
  • Physician's qualitative assessment of hemostasis [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
  • Adverse events
  • Changes in biochemical parameters indicating renal or hepatic impairment
  • Seroconversion to HIV/1, HIV/2, HAV, HBV, HCV or parvovirus B19 in subjects seronegative for these viruses at the time of enrollment
  • Amount of product used per year as part of at-home prophylaxis and therapy for bleeding episodes
  • Physician's qualitative assessment of hemostasis
Not Provided
Not Provided
 
Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
Phase IV A Study of Immunologic Safety for Alphanate in Previously Treated Patients Diagnosed With Severe Hemophilia A

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.

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.

Interventional
Phase 4
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Severe Hemophilia A
Drug: Alphanate SD/HT
Plasma-derived preparation of Factor VIII
Other Name: Anti-hemophilic (human) coagulation factor VIII
Experimental: Coagulation factor VIII (Human)
Anti-Hemophilic coagulation factor VIII (Human) Alphanate SD/HT
Intervention: Drug: Alphanate SD/HT
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
60
March 2020
December 2019   (final data collection date for primary outcome measure)

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).
Male
6 Years to 65 Years
No
Contact: Paul J Pinciaro, PhD 410-814-7617 paul.pinciaro@grifols.com
United States,   Poland
 
NCT00323856
GBI 04-01
No
Grifols Biologicals Inc.
Grifols Biologicals Inc.
Not Provided
Study Director: Paul J Pinciaro, PhD Grifols Biologicals Inc.
Grifols Biologicals Inc.
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP