Genetic Diseases clinical trials at UCSF
2 in progress, 1 open to eligible people
open to eligible males ages 5 years and up
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Oakland, California and other locations
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Sorry, in progress, not accepting new patients
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.
San Francisco, California and other locations