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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-005010-31 | EudraCT Number |
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A study to evaluate the effectiveness of Liposomal Amikacin for Inhalation (LAI) 590 mg administered once daily (QD) when added to multi-drug regimen (MDR) in participants with Nontuberculous Mycobacterial (NTM) lung infection caused by Mycobacterium Avium Complex (MAC) that were refractory to treatment.
Participants were randomized 2:1 to LAI 590 mg administered QD + MDR or MDR alone.
This is a randomized, open-label, multicenter study of LAI in adult participants with NTM lung infections caused by MAC that were refractory to treatment. Participants received either LAI 590 mg administered QD by inhalation plus a multidrug regimen, hereafter referred to as LAI + MDR or a multidrug regimen alone, hereafter referred to as MDR alone for a minimum of 8 months. Participants who demonstrated culture conversion by Month 6 went on to complete a treatment course of 12 months, starting from the first of 3 negative cultures that defined culture conversion.
Sputum culture results were made available to the site after the Month 6 sputum result was known, in time for the Month 8 visit. Prior to the Month 8 visit, the culture results from Baseline to Month 6 inclusively were blinded to the site and Sponsor. The results were blinded to reduce the potential for bias in an open-label study. At Month 8 (-28 to +7 days), after all sputum culture results were made available to the site only, up to and including Month 6, participants were assessed as converters or non-converters.
A converter was defined as a participant who had 3 consecutive monthly MAC-negative sputum cultures at any time within the first 6 months of the study.
"Relapse or recurrence" was defined as having MAC-positive sputum cultures in liquid broth media (agar negative) for 3 or more consecutive months, or having at least 1 MAC-positive sputum culture on solid media (agar positive) after achieving culture conversion.
A non-converter was defined as a participant who did not have 3 consecutive monthly MAC-negative sputum cultures at any time within the first 6 months of the study.
All converters remained in the study. Converters who, after culture conversion, subsequently had MAC-positive sputum cultures in liquid broth media (agar negative) for 1 or 2 consecutive months only by Month 6 also remained in the study. Participants who remained in the study continued their randomized treatment regimen until they completed a total of 12 months of treatment (EOT), starting from the first of 3 negative cultures that defined culture conversion. These participants returned after the EOT visit for 28 days, 3, 6, and 12 months off-treatment follow-up visits. The 12 months off-treatment follow-up visit was the end of study (EOS) visit. No NTM treatment was administered during the off-treatment phase.
At Month 8, all non-converters as assessed at the Month 6 visit were discontinued from Study INS-212. Participants who experienced a relapse or recurrence by Month 6 also discontinued from Study INS-212 at their Month 8 visit. These participants were potentially eligible to enter a separate open-label study of LAI (Study INS-312), provided all entry criteria were met for that study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Multi-drug Regimen | No Intervention | Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America [ATS/IDSA] Guidelines) | |
| LAI + Multi-drug Regimen | Experimental | Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LAI (Liposomal Amikacin for Inhalation) 590 mg | Drug | LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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. | by Month 6 |
| Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | 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. | up to Month 19 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gina Eagle, MD | Insmed Incorporated | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41186908 | Derived | Yamazaki Y, Nakagawa T, Jumadilova Z, Yuen D, Villa R, Hasegawa N. Management and Resolution of Hypersensitivity Pneumonitis-Related Events in Japanese Patients Treated with Amikacin Liposome Inhalation Suspension in the CONVERT and INS-312 Clinical Trials. Infect Dis Ther. 2026 Jan;15(1):365-379. doi: 10.1007/s40121-025-01247-7. Epub 2025 Nov 4. | |
| 38277865 |
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Participant flow table reflects the disposition through end of the study (Month 28).
The deaths under Reasons for Discontinuation reflect the primary reason (according to investigator's judgment) that the participant discontinued and does not represent all deaths in the trial.
This study was conducted at 127 sites in 18 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | LAI + Multi-drug Regimen | Participants received Liposomal Amikacin for Inhalation (LAI) 590 mg once daily (QD) in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America [ATS/IDSA] Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes |
| FG001 | Multi-drug Regimen | Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | LAI + Multi-drug Regimen | Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | 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. | 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. | Posted | Count of Participants | Participants | by Month 6 |
Treatment-emergent adverse events (AEs) were assessed at Baseline (Day 1) and all subsequent study visits through Month 28 (end of study).
The analysis population consisted of the safety population, defined as all participants who received at least 1 dose of either LAI+ MDR (multi-drug regimen) or MDR alone.
Participants counted in all-cause mortality constitutes all deaths, regardless of whether or not there was an adverse event.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | LAI + Multi-drug Regimen | Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kevin Mange (Senior Vice President, Clinical Development & Medical Affairs, ALIS) | Insmed Inc | 908-947-2651 | kevin.mange@insmed.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 22, 2016 | Oct 16, 2019 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 24, 2016 | Oct 16, 2019 | SAP_003.pdf |
| ID | Term |
|---|---|
| D009165 | Mycobacterium Infections, Nontuberculous |
| ID | Term |
|---|---|
| D009164 | Mycobacterium Infections |
| D000193 | Actinomycetales Infections |
| D016908 | Gram-Positive Bacterial Infections |
| D001424 | Bacterial Infections |
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| ID | Term |
|---|---|
| D001239 | Inhalation |
| ID | Term |
|---|---|
| D015656 | Respiratory Mechanics |
| D012119 | Respiration |
| D012143 | Respiratory Physiological Phenomena |
| D002943 | Circulatory and Respiratory Physiological Phenomena |
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|
| at Month 6 |
| Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm | 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. | by Month 6 |
| Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone | 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. | up to Month 16 |
| Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone | 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. | up to Month 16 |
| Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score | 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. | at Month 6 |
| Morimoto K, Nonaka M, Yamazaki Y, Nakagawa T, Takasaki J, Tsuyuguchi K, Kitada S, Jumadilova Z, Yuen DW, Ciesielska M, Hasegawa N. Amikacin liposome inhalation suspension for Mycobacterium avium complex pulmonary disease: A subgroup analysis of Japanese patients in the randomized, phase 3, CONVERT study. Respir Investig. 2024 Mar;62(2):284-290. doi: 10.1016/j.resinv.2023.12.012. Epub 2024 Jan 25. |
| 33887244 | Derived | Griffith DE, Thomson R, Flume PA, Aksamit TR, Field SK, Addrizzo-Harris DJ, Morimoto K, Hoefsloot W, Mange KC, Yuen DW, Ciesielska M, Wallace RJ Jr, van Ingen J, Brown-Elliott BA, Coulter C, Winthrop KL; CONVERT Study Group. Amikacin Liposome Inhalation Suspension for Refractory Mycobacterium avium Complex Lung Disease: Sustainability and Durability of Culture Conversion and Safety of Long-term Exposure. Chest. 2021 Sep;160(3):831-842. doi: 10.1016/j.chest.2021.03.070. Epub 2021 Apr 19. |
| 33595792 | Derived | Rubino CM, Onufrak NJ, van Ingen J, Griffith DE, Bhavnani SM, Yuen DW, Mange KC, Winthrop KL. Population Pharmacokinetic Evaluation of Amikacin Liposome Inhalation Suspension in Patients with Treatment-Refractory Nontuberculous Mycobacterial Lung Disease. Eur J Drug Metab Pharmacokinet. 2021 Mar;46(2):277-287. doi: 10.1007/s13318-020-00669-7. Epub 2021 Feb 17. |
| 30216086 | Derived | Griffith DE, Eagle G, Thomson R, Aksamit TR, Hasegawa N, Morimoto K, Addrizzo-Harris DJ, O'Donnell AE, Marras TK, Flume PA, Loebinger MR, Morgan L, Codecasa LR, Hill AT, Ruoss SJ, Yim JJ, Ringshausen FC, Field SK, Philley JV, Wallace RJ Jr, van Ingen J, Coulter C, Nezamis J, Winthrop KL; CONVERT Study Group. Amikacin Liposome Inhalation Suspension for Treatment-Refractory Lung Disease Caused by Mycobacterium avium Complex (CONVERT). A Prospective, Open-Label, Randomized Study. Am J Respir Crit Care Med. 2018 Dec 15;198(12):1559-1569. doi: 10.1164/rccm.201807-1318OC. |
| Physician Decision |
|
| Withdrawal by Subject |
|
| Rescue Medication |
|
| Other Not Specified |
|
| Protocol Violation |
|
| Multi-drug Regimen |
Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| If female, is participant of childbearing potential? | 165/224 female participants in the LAI + Multi-drug regimen arm 68/112 female participants in the Multi-drug regimen arm | Count of Participants | Participants |
|
| If not childbearing potential, specify | 152/224 female participants not of childbearing potential in the LAI + Multi-drug regimen arm 62/112 female participants not of childbearing potential in the Multi-drug regimen arm | Count of Participants | Participants |
|
| Region | Count of Participants | Participants |
|
| Multidrug regimen prior to enrollment | Count of Participants | Participants |
|
| Smoking status (includes e-cigarettes) | Count of Participants | Participants |
|
| Prior nebulized IV amikacin | Participants who had received prior nebulized IV amikacin | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | LAI + Multi-drug Regimen | Participants received LAI 590 mg once daily (QD) in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes |
| OG001 | Multi-drug Regimen | Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) |
|
|
|
| Primary | Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone | 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. | 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. | Posted | Count of Participants | Participants | up to Month 19 |
|
|
|
|
| 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 | 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. | 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. | Posted | Mean | Standard Deviation | meters | at Month 6 |
|
|
|
|
| Secondary | Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm | 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. | 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. | Posted | Number | 95% Confidence Interval | months | by Month 6 |
|
|
|
|
| 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 | 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. | 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. | Posted | Count of Participants | Participants | up to Month 16 |
|
|
|
| Secondary | Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone | 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. | Due to the widely varying timepoints that makeup the EOT visit, this was not considered an appropriate analysis and was not performed. | Posted | up to Month 16 |
|
|
| Secondary | Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | at Month 6 |
|
|
|
| 11 |
| 223 |
| 45 |
| 223 |
| 199 |
| 223 |
| EG001 | Multi-drug Regimen | Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) | 8 | 112 | 23 | 112 | 72 | 112 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Cardiac failure | Cardiac disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Cardiogenic shock | Cardiac disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Microvascular coronary artery disease | Cardiac disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumatosis intestinalis | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Adenocarcinoma gastric | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 22.0 | Systematic Assessment |
|
| Colon adenoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 22.0 | Systematic Assessment |
|
| Lung adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 22.0 | Systematic Assessment |
|
| Breast cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 22.0 | Systematic Assessment |
|
| Major depression | Psychiatric disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Cachexia | Metabolism and nutrition disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Spinal pain | Musculoskeletal and connective tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Peripheral vascular disorder | Vascular disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Shock | Vascular disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Performance status decreased | General disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Radius fracture | Injury, poisoning and procedural complications | MedDRA version 22.0 | Systematic Assessment |
|
| Computerised tomogram thorax abnormal | Investigations | MedDRA version 22.0 | Systematic Assessment |
|
| Urinary retention | Renal and urinary disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pulmonary cavitation | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Alveolitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Maxillary sinus pseudocyst | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Bronchial fistula | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Respiratory acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Infective exacerbation of chronic obstructive airways disease | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Mycobacterium avium complex infection | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Lung infection | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Infectious pleural effusion | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Lung infection pseudomonal | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumonia pseudomonal | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Scedosporium infection | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Superinfection bacterial | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Mycetoma mycotic | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Acute myocardial infarction | Cardiac disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Hypercapnic coma | Nervous system disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Exostosis | Musculoskeletal and connective tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Weight decreased | Investigations | MedDRA version 22.0 | Systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA version 22.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA version 22.0 | Systematic Assessment |
|
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA version 22.0 | Systematic Assessment |
|
Per the signed Investigator Agreement in the study protocol and protocol amendments, the PI agreed "not to originate or use the name of Insmed Incorporated, or study drug code in any publicity, news release, or other public announcement, written or oral, whether to the public, press, or otherwise, relating to this protocol, to any amendment to the protocol, or to the performance of this protocol, without the prior written consent of Insmed Incorporated."
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Naturally Sterile |
|
| Japan |
|
| Rest of World |
|
| Month 6 |
|
|
| Change from Baseline to Month 6 |
|
|
| SGRQ Total Score at Month 6: |
|
|
| Change from Baseline to Month 6: |
|
|