A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin For Inhalation (Arikace) in Patients with Recalcitrant Nontuberculous Mycobacterial Lung Disease

Nontuberculous mycobacteria (NTM) are organisms common in the soil and in environmental and potable water, and have been associated with lung disease in select patient groups. Treatment requires lengthy multi-drug regimens that can be poorly tolerated and are often not very effective, especially in patients with severe disease or in those who have failed prior treatment attempts. There have been very few clinical trials to support current treatment recommendations and no new drugs have been assessed for this disease in many years.

Amikacin is an established antibiotic that is effective against a variety of NTM. However, intravenous amikacin treatment is limited by its systemic route of administration. Arikace®, liposomal amikacin for inhalation, is a sustained-release formulation of amikacin encapsulated inside very small liposomal carriers (fat particles) designed for administration via inhalation. This formulation allows for targeting the drug to the lungs and the site of disease. The purpose of this study is to determine whether Arikace® is effective in treating recalcitrant nontuberculous mycobacterial lung disease. The safety and tolerability of Arikace® in this patient population will also be assessed.