Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics. The major challenges of current treatment regimens, such as the short half-life of hemophilia therapeutics with the need for frequent IV injections, encourage the current efforts for gene transfer therapy. This study will evaluate the safety and preliminary efficacy of ASC618, an AAV vector encoding B-domain deleted codon-optimized human factor VIII under a synthetic liver-directed promoter
• Male ≥18 years of age
• Severe or moderately severe hemophilia A (FVIII activity ≤ 2 IU/dL) as evidenced by
• medical history
• Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated
• days (exposure days)
• ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
• BMI ≤ 30
• Agree to use double-barrier contraceptive until at least 3 consecutive semen samples are negative after ASC-618 infusion