Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
Completed · Phase 2/Phase 3
Conditions studied: Hemophilia A
In brief
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.
Key facts
- Study ID
- NCT00157040
- Run by
- Baxalta now part of Shire
- People needed
- 50
- Starts
- 2002-06-07
- Expected to finish
- 2005-01-04
- Last updated by the study team
- 2021-04-27
Who can join
Age: any, up to 6. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Children´s Hospital Los Angeles — Los Angeles, California, United States
- Emory University, Department of Pediatrics — Atlanta, Georgia, United States
- Children´s Memorial Hospital — Chicago, Illinois, United States
- Comprehensive Bleeding Disorders Center — Peoria, Illinois, United States
- Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana, United States
- University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics — Iowa City, Iowa, United States
- University of Michigan Hemophilia Treatment Center — Ann Arbor, Michigan, United States
- Children´s Hospital of Michigan — Detroit, Michigan, United States
- Children´s Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- University of Texas Health Science Center — Houston, Texas, United States
- Hospital for Sick Children, Division of Hematology/Oncology — Toronto, Ontario, Canada
- University Pediatric Hospital — San Juan, Puerto Rico
Full record on ClinicalTrials.gov
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