A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH) · Persistent Anemia
Simple summaryAI-assisted
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Locations
Toronto General Hospital, Toronto, Ontario, Canada
St. Vincent Hospital - The Catholic University of Korea, Suwon, Gyeonggi-do, South Korea
The Catholic University of Korea, Seoul St. Mary's Hospital, Seoul, South Korea
Seoul National University Hospital, Seoul, South Korea
Severance Hospital, Seoul, South Korea
Samsung Medical Center, Seoul, South Korea
St. James's University Hospital, Leeds, West Yorkshire, United Kingdom
Kings College Hospital NHS Foundation Trust, London, United Kingdom
Study leads
Clinical Trial Management
Study Director
Regeneron Pharmaceuticals