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Anti-Infective Completed with Results
Arikace for Nontuberculous Mycobacteria (NTM) (Insmed 112)
This study looked at the safety and effectiveness of liposomal amikacin for inhalation (Arikace™), in treating nontuberculous mycobacterial lung disease (NTM).
Participants in this study included both people with CF and people without CF. To be included in the study, participants were required to have persistently positive respiratory cultures for NTM despite having received 6 months or more of standard antibiotic treatment for their NTM. The study had two parts. In the first part of the study, participants were randomized to receive once daily liposomal amikacin for inhalation (LAI) at 590mg or placebo, in addition to the other antibiotics that they were already receiving, for 84 days. In the second part of the study, all participants could receive LAI for an additional 84 days.
Eligibility
See other primary eligibility criteria for more information.
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Age:
12 Years to 75 Years -
Mutation(s):
No Mutation Requirement -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Subjects must have active non-tuberculous mycobacterial lung disease. This study includes CF and non-CF patients.
Study Results
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What We Learned:
The majority of participants in this study did not have CF, which made the results difficult to interpret for people with CF. Overall, while a trend showing reduced mycobacterial growth in the sputum of participants treated with LAI was observed, the reduction was not statistically significant. However, in individuals without CF, treatment with LAI resulted in significantly more participants having negative sputum cultures compared with placebo (p=0.006). The occurrence of hospitalizations and adverse events leading to drug discontinuation in the LAI group was greater than the occurrence of those events in the placebo group.
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Primary Findings:
Effectiveness:
This study was conducted between March 2012 and December 2015. Of the 89 participants randomized to receive treatment with LAI or placebo, 78 elected to continue in the open-label extension and 59 completed both parts of the study. Of the 89 total participants, 17 were people with cystic fibrosis. The primary efficacy endpoint was change in quantity of mycobacterial organisms in sputum from baseline to Day 84. While a trend showing reduced mycobacterial growth in participants treated with LAI was observed, the reduction was not statistically significant; thus this study did not meet its primary endpoint. However, significantly more participants, who did not have CF and received LAI compared with placebo, had negative sputum cultures at day 84 (32% vs. 9%, p=0.006).
Researchers also measured changes in lung function and conducted a 6-minute walk test at baseline and at the end of the study. The LAI group demonstrated a statistically significant improvement in the 6-minute walk test compared with the placebo group (mean of 45.6 meters greater distance vs. placebo (P=0.017)). No meaningful changes in lung function were observed.
Safety:
In the first part of the study, the overall occurrence of hospitalization for adverse events was higher in the LAI group compared with the placebo group (18.2% vs. 8.9%). Additionally, seven (15.9%) participants in the LAI group and none in the placebo group discontinued use of the study drug because of an adverse event. The most common reasons for study drug discontinuation were pulmonary exacerbation (3 participants) and difficult or labored breathing (dyspnea) (2 participants).
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Citation:
Am J Respir Crit Care Med 2016;[Epub ahead of print]
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
Yes -
Length of Participation:
12 weeks -
Number of Study Visits:
0
Additional Information
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Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Insmed -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
12 Years to 75 Years -
Mutation(s):
No Mutation Requirement -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Subjects must have active non-tuberculous mycobacterial lung disease. This study includes CF and non-CF patients.
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