Clinical Trial: Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional




Official Title: A Phase 1/2, Open-Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 (Recombinant AAV2/6 Human Factor 8 Gene Therapy) in Adult Subjects With Severe Hemophilia A

Brief Summary: The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity levels with adaptive doses of SB-525.