The trial is a multi-center, multi-national, randomized, double-blind, placebo-controlled study. The primary objective of the study is to assess the safety and tolerability of aerosolized ALN-RSV01 versus placebo in lung transplant patients naturally infected with respiratory syncytial virus (RSV). Secondary objectives include evaluating the anti-viral activity and pharmacokinetics of ALN-RSV01.
The trial is expected to enroll 21 patients who will be randomized in a two to one, drug to placebo ratio. All patients will receive standard of care, and those receiving ALN-RSV01 will have drug administered as a 0.6 mg/kg dose by inhalation via nebulizer once daily for three days.
Methods for measuring anti-viral activity include nasal swabs to determine level of viral shedding, as well as bronchoalveolar lavage (BAL) sampling in certain patients as determined necessary by the attending physician. BAL is a minimally invasive medical procedure that allows measurement of infection in the lung.
Akshay Vaishnaw, vice president, clinical research at Alnylam, said: “While our primary focus is on developing an RNAi therapeutic for pediatric RSV populations, we see a clear need for new treatments in certain adult patient settings and recognize the importance that comprehensive safety and exploratory efficacy bring to our overall ALN-RSV01 development program.”