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

NCT00323856 | Completed Phase 4 Results FDA Drug

• 1 other identifier: GBI 04-01
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 2

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.

Conditions

Severe Hemophilia A

Keywords

Hemophilia A; Plasma-derived treatment; Factor VIII; Inhibitor

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Factor VIII (FVIII) Inhibitor Development

  Incidence of FVIII inhibitor development was defined as any result determined positive at a central laboratory (inhibitor titer of greater than 0.6 modified Bethesda Units/milliliters \[BU/mL\]) using Nijmegen modification of the Bethesda assay.

  Up to Month 30

Secondary Outcomes (9)

• Number of Participants With Adverse Events (AE)

  Up to Month 30

• Change From Baseline in Alkaline Phosphatase

  Baseline and Quarterly Visit 1 (Month 3), 2 (Month 6), 3 (Month 9), 4 (Month 12), 5 (Month 15), 6 (Month 18), 7 (Month 21), 8 (Month 24), 9 (Month 27) and 10 (Month 30)

• Change From Baseline in Alanine Aminotransferase

  Baseline and Quarterly Visit 1 (Month 3), 2 (Month 6), 3 (Month 9), 4 (Month 12), 5 (Month 15), 6 (Month 18), 7 (Month 21), 8 (Month 24), 9 (Month 27) and 10 (Month 30)

• Change From Baseline in Aspartate Aminotransferase

  Baseline and Quarterly Visit 1 (Month 3), 2 (Month 6), 3 (Month 9), 4 (Month 12), 5 (Month 15), 6 (Month 18), 7 (Month 21), 8 (Month 24), 9 (Month 27) and 10 (Month 30)

• Change From Baseline in Lactate Dehydrogenase

  Baseline and Quarterly Visit 1 (Month 3), 2 (Month 6), 3 (Month 9), 4 (Month 12), 5 (Month 15), 6 (Month 18), 7 (Month 21), 8 (Month 24), 9 (Month 27) and 10 (Month 30)

Study Arms (1)

Coagulation factor VIII (Human)

EXPERIMENTAL

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

Drug: Alphanate SD/HT

Interventions

Alphanate SD/HT DRUG

Plasma-derived preparation of Factor VIII

Also known as: Anti-hemophilic (human) coagulation factor VIII

Coagulation factor VIII (Human)

Eligibility Criteria

• Age: 6 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Grifols Biologicals, LLC: lead

Study Sites (2)

Oddzial Chorob Wewnetrznych i Hematologii

Poznan, Szkolna, Poland

Location

Katedra i Klinika Hematologii Collegium Medicum UJ

Krakow, Poland

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Rhonda Griffin
• Organization: Grifols

rhonda.griffin@grifols.com

919-316-6693

Study Officials

• Michael Ken Woodward

  Instituto Grifols SA

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2006
• First Posted: May 10, 2006
• Study Start: April 8, 2003
• Primary Completion: December 11, 2018
• Study Completion: December 14, 2018
• Last Updated: January 16, 2025
• Results First Posted: June 9, 2022

Record last verified: 2025-01

Locations

PL (2)