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

NCT ID: NCT00323856

Last Updated: 2025-01-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 51 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-04-08
• Study Completion Date: 2018-12-14

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.

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.

Conditions

Severe Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Coagulation factor VIII (Human)

Anti-Hemophilic coagulation factor VIII (Human) Alphanate SD/HT

Group Type: EXPERIMENTAL

Alphanate SD/HT

Intervention Type: DRUG

Plasma-derived preparation of Factor VIII

Interventions

Alphanate SD/HT

Plasma-derived preparation of Factor VIII

Intervention Type: DRUG

Other Intervention Names

Anti-hemophilic (human) coagulation factor VIII

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).

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Grifols Biologicals, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael Ken Woodward

Role: STUDY_DIRECTOR

Instituto Grifols SA

Locations

Oddzial Chorob Wewnetrznych i Hematologii

Poznan, Szkolna, Poland

Katedra i Klinika Hematologii Collegium Medicum UJ

Krakow, , Poland

Countries

Poland

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

GBI 04-01

Identifier Type: -

Identifier Source: org_study_id