Nucleonics plans to enroll a total of 15 patients infected with hepatitis B virus (HBV) with mild to moderate disease and no evidence of cirrhosis at three US clinical sites and two sites in Eastern Europe. The patients are being organized into five escalating dose groups of three patients per group.
Primary endpoints are safety-related, with secondary endpoints tracking biological markers of efficacy, including HBV viral levels in the blood and circulating HBV surface antigen (HBsAg) levels. An independent safety monitor as well as a data safety monitoring board (DSMB) will oversee the trial. There is a planned stop and interim safety analysis after completion of the third dose group before proceeding to the fourth and fifth cohorts.
Robert Towarnicki, president and CEO of Nucleonics, said: “We are very pleased to initiate this clinical study, which represents one of the first systemic administrations of any RNAi-based therapeutic and is the first RNAi treatment directed against HBV.”