Hemophilia Inhibitor Previously Untreated Patient Study
- Conditions
- Hemophilia A
- Interventions
- Drug: FVIII concentrate
- Registration Number
- NCT01652027
- Brief Summary
Hemophilia A is a congenital bleeding disorder caused by deficiency of factor VIII (FVIII) and is treated by replacement therapy with FVIII concentrate. Approximately 30% of people with severe hemophilia A develop neutralizing antibodies, called FVIII inhibitors, which interfere with the function of FVIII concentrates. The reason that some, but not all, people with severe hemophilia A develop inhibitors is incompletely understood. Understanding individual and environmental risk factors is important to be able to prevent and possibly treat inhibitors. This study will look at individual and treatment characteristics in babies with severe hemophilia A who have not yet received treatment with FVIII (called Previously Untreated Patients, or PUPS). Subjects in the study will be asked to provide diaries of treatments, medications, and illnesses. Treatment will be directed by the subjects' physician, but all subjects will receive Advate, a third-generation recombinant FVIII product. Subjects will have blood drawn for laboratory tests, which include studies of the immune system and genetic studies of the FVIII mutation, before and 7-9 days after the first treatment with FVIII, and 5 days (+/-2 days) after the 5th, 10th, 20th, 30th, 40th, and 50th days of treatment with FVIII (exposure days). The duration of the study will be first 50 treatments or 3 years, whichever comes first.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 25
- Severe hemophilia A with FVIII activity < 1% normal
- Weight > 3.5 kg at the time of baseline study evaluation
- Informed consent, approved by appropriate Institutional Review Board/Independent Ethics Committee, has been administered, signed, and dated
- Prior exposure to clotting factor concentrates or blood products
- Other chronic disease
- Currently participating in another investigational drug study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Previously Untreated Patients with Hemophilia A FVIII concentrate -
- Primary Outcome Measures
Name Time Method Total number of FOXP3-positive T regulatory cells in the circulation 50 exposure days to FVIII or 3 years, whichever comes first FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.
- Secondary Outcome Measures
Name Time Method FVIII-specific T-cells 50 exposure days to FVIII or 3 years, whichever comes first FVIII-specific T-cells will be compared before and after exposure to FVIII
Trial Locations
- Locations (17)
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Tulane University
🇺🇸New Orleans, Louisiana, United States
Cornell University
🇺🇸New York, New York, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Medical University of Vienna
🇦🇹Vienna, Austria
Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
🇮🇹Milan, Italy
Malmo Centre for Thrombosis and Haemostasis
🇸🇪Malmo, Sweden
Emma Children's Hospital AMC
🇳🇱Amsterdam, Netherlands
Emory University
🇺🇸Atlanta, Georgia, United States
Hemophilia Center of Western Pennsylvania
🇺🇸Pittsburgh, Pennsylvania, United States
North Texas Comprehensive Hemophilia Center
🇺🇸Dallas, Texas, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Texas Health Science Center-Houston
🇺🇸Houston, Texas, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Indiana Hemophilia and Thrombosis Center
🇺🇸Indianapolis, Indiana, United States
Intermountain Hemophilia and Thrombosis Center
🇺🇸Salt Lake City, Utah, United States
University of Oregon
🇺🇸Portland, Oregon, United States