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

This study is currently recruiting participants. (see Contacts and Locations)
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
  Purpose

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 Intervention Phase
Severe Hemophilia A
Drug: Alphanate SD/HT
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Grifols Biologicals Inc.:

Primary Outcome Measures:
  • Incidence of Factor VIII Inhibitor Development [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 60
Study Start Date: January 2003
Estimated Study Completion Date: March 2020
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   6 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00323856

Contacts
Contact: Paul J Pinciaro, PhD 410-814-7617 paul.pinciaro@grifols.com

Locations
United States, Oklahoma
University of Oklahoma Children's Hospital Recruiting
Oklahoma City, Oklahoma, United States, 73126
United States, Texas
Gulf States Hemophilia and Thrombophilia Center Recruiting
Houston, Texas, United States, 77030
Poland
Oddzial Chorob Wewnetrznych i Hematologii Active, not recruiting
Poznan, Szkolna, Poland
Katedra i Klinika Hematologii Collegium Medicum UJ Recruiting
Krakow, Poland
Sponsors and Collaborators
Grifols Biologicals Inc.
Investigators
Study Director: Paul J Pinciaro, PhD Grifols Biologicals Inc.
  More Information

No publications provided

Responsible Party: Grifols Biologicals Inc.
ClinicalTrials.gov Identifier: NCT00323856     History of Changes
Other Study ID Numbers: GBI 04-01
Study First Received: May 8, 2006
Last Updated: February 17, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Grifols Biologicals Inc.:
Hemophilia A
Plasma-derived treatment
Factor VIII
Inhibitor

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 01, 2014