Regeneron for Hemophilia B
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Description
This research study is seeking participants who have hemophilia B and need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also control bleeding events. To join the study , you must be male as assigned at birth, and between the ages of 16 and 65 years at the time of signing the informed consent form. The purpose of this study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental drug or treatment being tested in this study. The study is informational, and part of a larger program to understand and treat hemophilia B with a potential experimental new therapy in the future. After participants complete this research study, should they be interested and eligible, they may be offered the opportunity to take part in a future study with this new therapy. There is no obligation to agree to taking part in this future study.
Eligibility and criteria
What to expect
The study consists of 1 pre-screening visit, 1 screening visit, and 6 visits during the study period. The pre-screening portion of the study involves blood tests and medical record review. The study will collect blood to test for antibodies to AAV8, a type of virus vehicle used to deliver gene insertion therapy. There will be medical record review throughout the study to collect information about medical history, current health, diagnosis, treatments, medications, and results of clinical tests. The main study involves medical record review, blood tests, vital signs, physical examination, FibroScan and Liver Ultrasound, Hemophilia Joint Health Exam, usage of an electronic diary, completion of questionnaires, and testing for infection.
Related specialties
Related conditions
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