Trial · NCT02344004
Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone
NCT02344004, Phase 3, Completed, Insmed Incorporated; results first posted 2018-11-20
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone. Trials, record NCT02344004. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02344004
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone
- NCT number
- NCT02344004
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 336 participants, actual
- Lead sponsor
- Insmed Incorporated
- Conditions
- Mycobacterium Infections, Nontuberculous
- Interventions
- LAI (Liposomal Amikacin for Inhalation) 590 mg
- First posted
- 2015-01-22
- Last updated
- 2020-05-07
- Other registries' IDs
- eudract:2014-005010-31
- Results first posted
- 2018-11-20
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | by Month 6 | Sputum specimens were collected at Screening (Visit 1), Baseline (Visit 2), and at Visits 3 (Month 1) through 8 (Month 6). A negative culture result reflected a negative culture result for all sputum samples collected at each visit. Participants met the primary endpoint of culture conversion by Month 6 if they had 3 consecutive monthly MAC-negative sputum cultures during the first 6 months of the study. A participant needed to achieve the first of 3 consecutive negative sputum cultures (that defined culture conversion) by Month 4 in order to meet the primary endpoint by Month 6. Each participant in the intent to treat (ITT) population (ie, all randomized participants) was classified as either a converter or non-converter by Month 6. |
| Primary | Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | up to Month 19 | Sputum specimens were collected at screening, baseline (Day 1), during treatment, and at Months 1, 3, 6, and 12 months off treatment. Culture conversion with durability was defined as achieving culture conversion by Month 6 and then having no more than 2 consecutive broth positive cultures and no Agar positive culture up to 3 months off treatment. Converters with missing broth or Agar sputum culture result after Month 6 up to 3 months off treatment were considered as not achieving culture conversion with durability except those participants who are unable to produce sputum despite reasonable efforts, as reported by source documentation. Participants who had relapse/recurrence, had "rescue" medication and/or died before reaching 3 months off treatment were considered as not achieving culture conversion with durability. |
| Secondary | Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm | at Month 6 | A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and at Month 6. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment. The analysis of the change from Baseline (Day 1) to Month 6 in the 6MWT distance was performed after the last participant completed Month 6 and his/her 6MWT distance data were available. |
| Secondary | Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm | by Month 6 | The time to culture conversion was defined by the date of the first of at least 3 consecutive monthly culture specimens that were Mycobacterium avium complex (MAC)-negative. The 25th percentile time to conversion is the estimated time taken for 25% of participants to convert. The 50th percentile time to conversion is the estimated time taken for 50% of participants to convert. |
| Secondary | Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone | up to Month 16 | Sustained conversion was evaluated in participants who completed at least 12 months of treatment from the start of culture conversion. Sustained conversion was defined as conversion (3 consecutive negative monthly sputum samples) by Month 6 with no positive agar media culture or no more than 2 broth media cultures up to and including the time point. Participants who did not convert were considered non-sustained conversions. |
| Secondary | Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone | up to Month 16 | A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and up to EOT or Month 16. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment. |
| Secondary | Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score | at Month 6 | The SGRQ was completed before administration of study drug at Baseline (Day 1) and Months 3, 6, 8, and 12, and at the EOT visit and the 3 months off treatment visit. The SGRQ is a self-administered questionnaire that has been validated in participants with airways disease, specifically in participants with bronchiectasis. The SGRQ assesses health-related quality of life in participants with chronic pulmonary disease by evaluating 3 health domains: symptoms (distress caused by respiratory symptoms); activity (effects of disturbances on mobility and physical activity); and impacts (the effect of disease on factors such as employment, personal control of one's health, and need for medication). A composite total score is derived as the sum of domain scores for symptoms, activity, and impact (0 = best possible score and 100 = worst possible score). A within patient reduction from baseline in score of 4 units is generally recognized as a clinically meaningful improvement in quality of life. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | by Month 6 | The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | up to Month 19 | The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm | at Month 6 | The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | meters | MEAN |
| SECONDARY | Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm | by Month 6 | There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | months | NUMBER |
| SECONDARY | Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone | up to Month 16 | The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone | up to Month 16 | Due to the widely varying timepoints that makeup the EOT visit, this was not considered an appropriate analysis and was not performed. | ||
| SECONDARY | Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score | at Month 6 | The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | LAI + Multi-drug Regimen | Converter | 65 | Participants | |
| Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | Multi-drug Regimen | Converter | 10 | Participants | |
| Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | LAI + Multi-drug Regimen | Non-Converter | 159 | Participants | |
| Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm | Multi-drug Regimen | Non-Converter | 102 | Participants | |
| Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | LAI + Multi-drug Regimen | Durable Conversion | 36 | Participants | |
| Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | Multi-drug Regimen | Durable Conversion | 0 | Participants | |
| Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | LAI + Multi-drug Regimen | Non-Durable Conversion | 188 | Participants | |
| Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | Multi-drug Regimen | Non-Durable Conversion | 112 | Participants | |
| Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm | LAI + Multi-drug Regimen | Baseline | 424.2 | Standard Deviation 1.12 | meters |
| Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm | Multi-drug Regimen | Baseline | 420.9 | Standard Deviation 0.48 | meters |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | LAI + Multi-drug Regimen | 224 | |
| Overall Study | STARTED | Multi-drug Regimen | 112 | |
| Overall Study | COMPLETED | LAI + Multi-drug Regimen | 163 | |
| Overall Study | COMPLETED | Multi-drug Regimen | 98 | |
| Overall Study | NOT COMPLETED | LAI + Multi-drug Regimen | 61 | |
| Overall Study | NOT COMPLETED | Multi-drug Regimen | 14 |
Source ClinicalTrials.gov
Adverse events174
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 | Dysphonia | Respiratory, thoracic and mediastinal disorders | LAI + Multi-drug Regimen | 104 | 223 | 140 |
| other | Dysphonia | Respiratory, thoracic and mediastinal disorders | Multi-drug Regimen | 2 | 112 | 2 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | LAI + Multi-drug Regimen | 85 | 223 | 111 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Multi-drug Regimen | 17 | 112 | 20 |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | LAI + Multi-drug Regimen | 47 | 223 | 59 |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | Multi-drug Regimen | 10 | 112 | 10 |
| other | Haemoptysis | Respiratory, thoracic and mediastinal disorders | LAI + Multi-drug Regimen | 37 | 223 | 48 |
| other | Haemoptysis | Respiratory, thoracic and mediastinal disorders | Multi-drug Regimen | 14 | 112 | 17 |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | LAI + Multi-drug Regimen | 24 | 223 | 30 |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Multi-drug Regimen | 2 | 112 | 2 |
1 to 10 of 174Source ClinicalTrials.gov
See all 174 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LAI + Multi-drug Regimen | 11 | 223 |
| Total, all-cause mortality | Multi-drug Regimen | 8 | 112 |
| Total, other adverse events | LAI + Multi-drug Regimen | 199 | 223 |
| Total, other adverse events | Multi-drug Regimen | 72 | 112 |
| Total, serious adverse events | LAI + Multi-drug Regimen | 45 | 223 |
| Total, serious adverse events | Multi-drug Regimen | 23 | 112 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
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 + Multi-drug Regimen vs Multi-drug Regimen: deaths from any cause | Trial comparisons |
| LAI + Multi-drug Regimen vs Multi-drug Regimen: serious adverse events | 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.
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/NCT02344004
GET https://conductscience.org/api/trials/v1/NCT02344004/results?page=2