Hemophilia A
Conditions
Keywords
Factor VIII Deficiency
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.
Interventions
Sponsors
Baxalta now part of Shire
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 6 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM) | 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 | Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model. |
Countries
Canada, Puerto Rico, United States
Outcome results
None listed