Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients

Brief Title

Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients

Official Title

Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (rAHF-PFM): Evaluation of Immunogenicity, Efficacy, and Safety in Previously Untreated Patients With Hemophilia A

Brief Summary

      The purpose of this study is to evaluate whether Antihemophilic factor, recombinant,
      manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A
      patients who have not been treated with factor VIII (FVIII) before.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Factor VIII Inhibitor Development

Secondary Outcome

 Bleeding Episodes Treated With 1 to ≥4 Infusions

Condition

Hemophilia A

Intervention

Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)

Study Arms / Comparison Groups

 Single Arm - All Participants

Description:  All subjects enrolled in the study who meet the eligibility criteria.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

April 1, 2004

Completion Date

September 11, 2009

Primary Completion Date

September 11, 2009

Eligibility Criteria

        Inclusion Criteria:

          -  The subject has severe or moderately severe hemophilia A as defined by a baseline
             factor VIII level <= 2% of normal, as documented at screening

          -  The subject is < 6 years of age

          -  The subject's legally authorized representative has provided written informed consent

        Exclusion Criteria:

          -  The subject has a history of exposure to factor VIII other than rAHF PFM or more than
             3 infusions of commercially available rAHF PFM (i.e., ADVATE) within 28 days prior to
             screening, as determined by the subject's medical history. Any infusion of factor VIII
             replacement products prior to the 28-day period excludes the subject from
             participation

          -  The subject has received more than 3 infusions of rAHF PFM (commercially available
             and/or study product) between screening and prior to the initial recovery infusion

          -  The subject has a detectable inhibitor to factor VIII, as measured in the screening
             sample by the Nijmegen assay in the central laboratory

          -  The subject has a history of inhibitor to factor VIII at any time prior to screening

          -  The subject has a known hypersensitivity to rAHF PFM

          -  The subject has any 1 of the following laboratory abnormalities at the time of
             screening:

               1. Platelet count < 100,000/mm^3

               2. Hemoglobin concentration < 10 g/dL (100 g/L)

               3. Serum creatinine > 1.5 times the upper limit of normal (ULN) for age

               4. Total bilirubin > 2 times the ULN for age

          -  The subject has an inherited or acquired hemostatic defect other than hemophilia A
             (e.g., qualitative platelet defect or von Willebrand's disease)

          -  The subject is known to be seropositive for human immunodeficiency virus (HIV),
             hepatitis C virus (HCV), or hepatitis B virus (HBV), as determined by the subject's
             medical history

          -  At the time of enrollment, the subject has a clinically significant chronic disease
             other than hemophilia A

          -  The subject is currently participating in another investigational drug study, or has
             participated in any clinical study involving an investigational drug within 120 days
             of the screening visit

          -  The subject (or the subject's legally authorized representative) is identified by the
             investigator as being unable or unwilling to cooperate with study procedures

          -  The subject has received any blood product, including packed red blood cells (RBC),
             platelets, plasma, or cryoprecipitate

Gender

All

Ages

N/A - 6 Years

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

Austria

Location Countries

Austria

Administrative Informations

NCT ID

NCT00157157

Organization ID

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Study Sponsor

Study Director, Study Director, Shire

Verification Date

May 2019