Mycobacterium Infections, Nontuberculous Clinical Trial
Official title:
A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin for Inhalation in Patients With Recalcitrant Nontuberculous Mycobacterial Lung Disease
The purpose of this study is to evaluate the efficacy, safety and tolerability of 84 days of
daily dosing of 590 mg of LAI versus placebo in patients with treatment refractory NTM lung
disease.
The first part of the study is the 84-day double-blind phase to evaluate the primary and
secondary endpoints.
This was a Phase 2, randomized, double-blind study of efficacy, safety, and tolerability of
once daily (QD) dosing of LAI 590 mg versus placebo for 84 days in subjects with treatment
refractory NTM lung infection on a stable multidrug regimen. At the conclusion of the
randomized double-blind phase of the study, subjects who consented to continue in the
open-label phase of the study received LAI 590 mg QD for 84 additional days. All subjects
were required to complete a 28-day safety follow-up after their end of treatment study visit
(Day 168). Subjects completing this study were consented for enrollment in a long-term
follow-up phase and were asked to return to the study site at 12 months and 24 months (per
protocol Amendment #3, the 24-month follow-up visit was no longer required) (visit window ± 2
months) after the last dose of study drug (either after completing the randomized
double-blind phase or the open-label phase). Subjects were stratified upon entering the study
based on the presence or absence of cystic fibrosis (CF) and Mycobacterium avium complex
versus M abscessus as the predominate NTM organism at baseline and were block randomized to
receive either LAI or placebo in a 1:1 ratio in the double-blind phase.
Subjects completing the main study, and subjects who completed the 84-day open-label phase,
were consented for enrollment in a post-LAI safety follow-up assessment and were asked to
return to the study site 12 months (visit window ± 2 months) after the last dose of study
drug (either after completing the randomized double-blind phase or the open-label phase). The
screening period required obtaining 3 morning sputum specimens (spontaneous or induced) for
mycobacteriology. At each post-screening sputum timepoint, at least 2 and preferably 3 sputum
specimens were obtained. At Day 1 (baseline), subjects were evaluated pre-dose and post-dose
at the study site. Subjects returned to the study site briefly on Day 2 for collection of
serum samples to determine creatinine clearance. On Days 28, 56, and 84, study drug was
administered at the site and the efficacy, safety, and tolerability of study drug were
evaluated. At Day 85, those subjects continuing in the open-label phase of the study received
LAI 590 mg at the study site with pre-dose and post-dose assessments for safety and efficacy.
Subjects returned every 28 days (Days 112, 140, and 168) in the open-label phase. A 28-day,
post-dose follow-up visit occurred at either Day 112 for those subjects who did not continue
in the open-label phase or at Day 196 for those subjects who continued in the open-label
phase.
Subjects completing the main study, and subjects who completed the 84-day open-label phase,
were consented for enrollment in a post-LAI safety follow-up assessment and were asked to
return to the study site 12 months (visit window ± 2 months) after the last dose of study
drug (either after completing the randomized double-blind phase or the open-label phase).
Arikace™, Arikayce™, Liposomal Amikacin for Inhalation (LAI), and Amikacin Liposome
Inhalation Suspension (ALIS) may be used interchangeably throughout this study and other
studies evaluating amikacin liposome inhalation suspension.
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