The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.
Study Type
OBSERVATIONAL
Enrollment
25
There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.
University of California San Francisco
San Francisco, California, United States
University of Florida
Gainesville, Florida, United States
Emory University
Atlanta, Georgia, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
Bloodworks Northwest
Seattle, Washington, United States
The Alfred Hospital
Melbourne, Victoria, Australia
Fiona Stanley Hospital
Murdoch, Western Austrailia, Australia
Providence Hematology/St. Paul's Hosptial
Vancouver, British Columbia, Canada
McMaster University / Royal Prince Alfred Hospital
Hamilton, Ontario, Canada
...and 1 more locations
Number of bleeding events, annualized
Annualized bleeding rate (ABR)
Time frame: 12 months
Dose and total FVIIII consumption
Total FVIII replacement therapy consumption and the corresponding dose
Time frame: 12 months
Annualized number of infusions (AIR)
Number of reported infusions over the study period
Time frame: 12 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.