The first clinical trial utilizing Aclarubicin for treatment of RVCL commenced December 5, 2016 at Washington University School of Medicine. Two patients initially received infusions of Aclarubicin for four consecutive days with blood work and other testing prior to and at the conclusion of the infusion. The study will be conducted for up to two years with patients participating in monthly cycles of five consecutive days per week (Day 1 for testing to verify the patient can participate followed by four days of infusions).
This clinical trial followed nearly a year of preparations including approval by the Food & Drug Administration for our Investigational New Drug application (IND # 131473, approved 8/2/16) entitled, “A Pilot Study of Aclarubicin for the Treatment of Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL).”