Trial · NCT01315236

Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria

NCT01315236, Phase 2, Completed, Insmed Incorporated; results first posted 2019-08-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria. Trials, record NCT01315236. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01315236

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria
NCT number: NCT01315236
Status: Completed
Phases: Phase 2
Enrollment: 90 participants, actual
Lead sponsor: Insmed Incorporated
Conditions: Mycobacterium Infections, Nontuberculous
Interventions: Liposomal amikacin for inhalation (LAI), placebo
First posted: 2011-03-15
Last updated: 2019-08-21
Results first posted: 2019-08-21

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Baseline and end of double-blind phase of 84 days	The endpoint used the 7-step semi-quantitative scale (SQS) for mycobacterial culture reporting in both solid and liquid growth media, with step 1 = culture negative in both solid and liquid media, step 2 = growth in liquid medium only, 3 = solid medium positive, 4 = 50 to 100 colonies in solid medium \& growth in liquid, 5 = \>100 to 200 colonies in solid medium \& growth in liquid, 6 = \>200 to 500 colonies in solid medium \& growth in liquid, 7 = \>500 colonies in solid medium \& growth in liquid. Full scale range is 1 (best score) to 7 (worst score). The change in step measures the growth at Day 84 compared to the growth at Baseline. The negative values represent reduction in colony growth.
Secondary	Number of Subjects With Negative NTM Culture for the LAI Arm at Day 84 Compared to the Placebo Arm at Day 84	84 days double-blind phase	Sputum specimens were cultured in liquid media in addition to solid media (agar). If results were negative on agar, the liquid media was held for 6 weeks before reporting as culture negative. Culture was negative when confirmed with no growth in liquid medium.
Secondary	Time to Negative NTM Culture….During the 84-day Double-blind Treatment Phase	84 days double-blind phase	Sputum specimens were cultured in liquid media in addition to solid media (agar). If results were negative on agar, the liquid media was held for 6 weeks before reporting as culture negative. Culture was negative when confirmed with no growth in liquid medium.
Secondary	Ordinal, 3-level Response From Baseline on the SQS for Mycobacterial Culture for the LAI Arm at Day 84 Compared to the Placebo Arm at Day 84	Baseline and end of double-blind phase of 84 days	The ordinal, 3-level response are (1) improvement (2) no change (3) worsening or death
Secondary	Change From Baseline in Respiratory and Systemic Symptoms Questionnaire (RSSQ) Score at Day 84 for the LAI Arm Compared to the Placebo Arm	Baseline to day 84.	The RSSQ was administered to gather information from the subject about the types of symptoms that the subject has experienced since the last contact. A reduction in score indicates improvement. The range of values for the scores are -2 (best) to +2 (worst) in whole numbers. The composite score was calculated by averaging the scores of the subscales.
Secondary	Change From Baseline in Global Rating of Health (GRH) at Day 84 for the LAI Arm Compared to the Placebo Arm	Baseline and end of double-blind phase of 84 days	The assessing physician asked the subject to rate his/her assessment of health according to the GRH. Subject responses to, "How would you rate your health at the present time?" included: Excellent, Good, Fair, or Poor.
Secondary	Number of Participants Requiring "Rescue" Anti-mycobacterial or Other "Rescue" Drugs During the 84-day Double-blind Phase	84 days double-blind phase	Per the study protocol, study subjects were on a stable, multi-drug, anti-mycobacterial regimen based on the 2007 ATS/IDSA Guidelines; the regimen should not have changed during the study period except for safety concerns. The need for changes to the concurrent anti-mycobacterial regimen or "rescue" therapy was at the discretion of the Investigator and was tracked as a study outcome.
Secondary	Number of Subject for "Rescue" Anti-mycobacterial or Other "Rescue" Drugs During the 84-day Double-blind Phase	84 days double-blind phase	Per the study protocol, study subjects were on a stable, multi-drug, anti-mycobacterial regimen based on the 2007 ATS/IDSA Guidelines; the regimen should not have changed during the study period except for safety concerns. The need for changes to the concurrent anti-mycobacterial regimen or "rescue" therapy was at the discretion of the Investigator and was tracked as a study outcome.

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Baseline and end of double-blind phase of 84 days	mITT	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Subjects With Negative NTM Culture for the LAI Arm at Day 84 Compared to the Placebo Arm at Day 84	84 days double-blind phase	mITT	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Time to Negative NTM Culture….During the 84-day Double-blind Treatment Phase	84 days double-blind phase	mITT	days	MEDIAN
SECONDARY	Ordinal, 3-level Response From Baseline on the SQS for Mycobacterial Culture for the LAI Arm at Day 84 Compared to the Placebo Arm at Day 84	Baseline and end of double-blind phase of 84 days	mITT	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline in Respiratory and Systemic Symptoms Questionnaire (RSSQ) Score at Day 84 for the LAI Arm Compared to the Placebo Arm	Baseline to day 84.	mITT	units on a score	MEAN
SECONDARY	Change From Baseline in Global Rating of Health (GRH) at Day 84 for the LAI Arm Compared to the Placebo Arm	Baseline and end of double-blind phase of 84 days	mITT~Subjects with missing data were excluded	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Participants Requiring Rescue Anti-mycobacterial or Other Rescue Drugs During the 84-day Double-blind Phase	84 days double-blind phase	mITT	participants	NUMBER
SECONDARY	Number of Subject for Rescue Anti-mycobacterial or Other Rescue Drugs During the 84-day Double-blind Phase	84 days double-blind phase	mITT	participants	NUMBER

Source ClinicalTrials.gov

Results by group104

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	LAI 590 mg QD	Baseline: Culture negative	5	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Placebo	Baseline: Culture negative	5	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	LAI 590 mg QD	Baseline: Growth in liquid medium only	1	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Placebo	Baseline: Growth in liquid medium only	1	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	LAI 590 mg QD	Baseline: Agar positive	17	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Placebo	Baseline: Agar positive	10	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	LAI 590 mg QD	Baseline: 1+ (50-100 colonies)	2	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Placebo	Baseline: 1+ (50-100 colonies)	4	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	LAI 590 mg QD	Baseline: 2+ (> 100-200 colonies)	2	Participants
Change in Semi-Quantitative Mycobacterial Culture Results From Baseline to Day 84.	Placebo	Baseline: 2+ (> 100-200 colonies)	2	Participants

1 to 10 of 104Source ClinicalTrials.gov

See all 104 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-blind Phase	STARTED	LAI 590 mg QD	44
Double-blind Phase	STARTED	Placebo	45
Double-blind Phase	COMPLETED	LAI 590 mg QD	40
Double-blind Phase	COMPLETED	Placebo	45
Double-blind Phase	NOT COMPLETED	LAI 590 mg QD	4
Double-blind Phase	NOT COMPLETED	Placebo	0
Open-label Phase	STARTED	LAI 590 mg QD	35
Open-label Phase	STARTED	Placebo	43
Open-label Phase	COMPLETED	LAI 590 mg QD	34
Open-label Phase	COMPLETED	Placebo	41

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events184

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Ear pain	Ear and labyrinth disorders	LAI 590 mg QD - Double Blind	3	44	3
other	Ear pain	Ear and labyrinth disorders	Placebo - Double Blind	0	45	0
other	Ear pain	Ear and labyrinth disorders	LAI 590 mg QD - Open Label	3	35	4
other	Ear pain	Ear and labyrinth disorders	Placebo - Open Label	0	43	0
other	Nausea	Gastrointestinal disorders	LAI 590 mg QD - Double Blind	5	44	5
other	Nausea	Gastrointestinal disorders	Placebo - Double Blind	4	45	4
other	Nausea	Gastrointestinal disorders	LAI 590 mg QD - Open Label	3	35	3
other	Nausea	Gastrointestinal disorders	Placebo - Open Label	5	43	6
other	Abdominal discomfort	Gastrointestinal disorders	LAI 590 mg QD - Double Blind	3	44	3
other	Abdominal discomfort	Gastrointestinal disorders	Placebo - Double Blind	0	45	0

1 to 10 of 184Source ClinicalTrials.gov

See all 184 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	LAI 590 mg QD - Double Blind	1	44
Total, all-cause mortality	Placebo - Double Blind	0	45
Total, all-cause mortality	LAI 590 mg QD - Open Label	1	35
Total, all-cause mortality	Placebo - Open Label	0	43
Total, other adverse events	LAI 590 mg QD - Double Blind	41	44
Total, other adverse events	Placebo - Double Blind	39	45
Total, other adverse events	LAI 590 mg QD - Open Label	31	35
Total, other adverse events	Placebo - Open Label	42	43
Total, serious adverse events	LAI 590 mg QD - Double Blind	8	44
Total, serious adverse events	Placebo - Double Blind	4	45

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
27748623	DERIVED	PubMed
33595792	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
LAI 590 mg QD vs Placebo: Number of Subjects With Negative NTM Culture for the LAI Arm at Day 84 Compared to the Placebo Arm at Day 84	Trial comparisons
LAI 590 mg QD vs Placebo: Number of Participants Requiring Rescue Anti-mycobacterial or Other Rescue Drugs During the 84-day Double-blind Phase	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01315236

GET https://conductscience.org/api/trials/v1/NCT01315236/results?page=2

API reference For AI assistants