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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Antihemophilic factor, recombinant, manufactured protein-free


Participation Criteria
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Eligibility Criteria
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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: platelet count < 100,000/mm3 hemoglobin concentration < 10 g/dL (100 g/L) serum creatinine > 1.5 times the ULN for age 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

Ages Eligible for Study
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Maximum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Antihemophilic factor, recombinant, manufactured protein-free|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM) | Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model. | Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Factor VIII Deficiency