Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A

Brief Title

Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A

Official Title

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

Brief Summary

      The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the
      treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study
      is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.

Study Phase

Phase 2/Phase 3

Study Type

Interventional

Primary Outcome

The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM)

Condition

Hemophilia A

Intervention

Antihemophilic factor, recombinant, manufactured protein-free

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

Drug

Estimated Enrollment

Start Date

June 7, 2002

Completion Date

January 4, 2005

Primary Completion Date

January 4, 2005

Eligibility Criteria

        Inclusion Criteria:

          -  Subject is less than 6 years of age

          -  Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level <=
             2%, documented at screening or on the basis of historical data (e.g., at hemophilia
             diagnosis)

          -  Documented medical history of at least 50 exposure days for treatment with all other
             factor VIII products

          -  Subject's parent or legally authorized representative has provided informed consent

        Exclusion Criteria:

          -  Detectable inhibitor to factor VIII measured in the screening sample by the local or
             central hemostasis laboratory

          -  History of inhibitor to factor VIII at any time prior to screening

          -  Subject has any one of the following laboratory abnormalities at the time of
             screening:

               1. platelet count < 100,000/mm3

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

               3. serum creatinine > 1.5 times the ULN for age

               4. total bilirubin > 2 times the ULN for age

          -  Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g.,
             platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)

          -  Subject has known hypersensitivity to RECOMBINATE rAHF

          -  Subject is currently participating in another investigational drug study or has
             participated in any clinical study involving an investigational drug within 30 days of
             study entry

          -  Subject is identified by the investigator as being unable or unwilling to cooperate
             with study procedures

Gender

All

Ages

N/A - 6 Years

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

Canada

Location Countries

Canada

Administrative Informations

NCT ID

NCT00157040

Organization ID

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Study Sponsor

Study Director, Study Director, Takeda

Verification Date

April 2021