Trial · NCT04677543
Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex
NCT04677543, Phase 3, Completed, Insmed Incorporated; results first posted 2024-06-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex. Trials, record NCT04677543. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04677543
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex
- NCT number
- NCT04677543
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 99 participants, actual
- Lead sponsor
- Insmed Incorporated
- Conditions
- Mycobacterium Infections, Nontuberculous
- Interventions
- ALIS, Azithromycin, Ethambutol, ELC
- First posted
- 2020-12-21
- Last updated
- 2024-06-28
- Other registries' IDs
- eudract:2020-002545-42
- Results first posted
- 2024-06-28
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Psychometric Cross-Sectional Validation of Patient Reported Outcome (PRO): Patient Global Impression of Severity (PGI-S) Respiratory Scale Score at Baseline | Baseline | The PGI-S respiratory symptom is a self-reported scale to measure the severity of illness based on symptoms using a 5-point scale ranging from 1 to 5, (1=not at all, 2=mild, 3=moderate, 4=very severe, 5=extremely severe). Considering different aspects of breathing symptoms like congestion, cough, mucus, wheezing, shortness of breath, participants rated their symptom severity on the PGI-S respiratory symptom scale. Higher scores indicate greater symptom severity. |
| Primary | Psychometric Cross-Sectional Validation of PRO: PGI-S Fatigue Scale Score at Baseline | Baseline | The PGI-S fatigue is a self-reported scale to measure the severity of illness based on symptoms using a 5-point scale ranging from 1 to 5, (1=not at all, 2=mild, 3=moderate, 4=very severe, 5=extremely severe). Participants rated the severity of their fatigue on the PGI-S fatigue scale. Higher scores indicate greater fatigue severity. |
| Primary | Psychometric Cross-Sectional Validation of PRO: Quality of Life Questionnaire - Bronchiectasis (QoL-B) Respiratory Symptoms Scale Score at Baseline | Baseline | The QOL-B is a self-administered, PRO questionnaire used to assess symptoms, functioning, and health related quality of life in adults with lung conditions. The respiratory symptom domain of the QOL-B contains 9 items describing patient's self-assessment of her/his respiratory symptoms that affect daily life. For each of the 8 items (chest congestion, coughing, cough up mucus, shortness of breath with greater activity, wheezing, chest pain, shortness of breath when talking, woken up during night due to cough), scores ranged from 1 to 4 (1= lot, 2= moderate, 3= little, 4= not at all) and the sputum item based on the color ranged from 0=don't know,1=green with traces of blood/brownish dark,2=yellowish-green,3=clear to yellow,4=clear. The item scores were summed and then standardized on a 0 to 100-point scale to derive the domain score with higher scores representing fewer symptoms or better functioning and quality of life. |
| Primary | Psychometric Cross-Sectional Validation of PRO: Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Score at Baseline | Baseline | The PROMIS F-SF 7a is a self-administered questionnaire assessing a range of self-reported symptoms over the past 7 days, from mild subjective feelings of tiredness to an overwhelming, debilitating, and sustained sense of exhaustion that likely decreases one's ability to execute daily activities and function normally in family or social roles. Fatigue is divided into the experience of fatigue (frequency, duration, and intensity) and the impact of fatigue on physical, mental, and social activities over 7 items. Response options are on a 5-point Likert scale, ranging from 1=never to 5=always. Total scores range from 7 to 35 and low scores represent less fatigue interference i.e., better symptoms. |
| Primary | Assessment of Test-Retest Reliability (TRTR) Reported as the Intraclass Co-relation (ICC) Estimate Among Participants Reporting no Change on Respiratory PGI-S Score Applied to QOL-B Respiratory Domain Score Between Screening and Baseline | From Screening to Baseline (Day -70 to Day 1) | TRTR consists of measuring the degree to which an instrument yield reproducible score at different points in time assessed across a fixed and common time interval for all subjects. TRTR co-relations were based on the two-way mixed effect ICC coefficient estimated using the inter-rater reliability package, version 0.84.1. TRTR estimate of 0.7 and above indicated better retest reliability. TRTR was assessed among participants reporting no change on PGI-S between screening and baseline. PGI-S anchors are PRO specific, with a respiratory PGI-S (scale ranging from 1=not at all to 5=extremely severe, Higher scores=greater symptom severity) applied to the QOL-B respiratory domain (9-item scale ranging from 0 to 100, higher scores=fewer symptoms and better quality of life). As pre-specified in statistical analysis plan(SAP) for participants contributing to this outcome measure from INS-415 study,data were collected and analyzed for combined population in which ALIS and ELC groups were pooled. |
| Primary | Assessment of TRTR Reported as the ICC Estimate Among Participants Reporting no Change on Fatigue PGI-S Score Applied to PROMIS F-SF 7a Score Between Screening and Baseline | From Screening to Baseline (Day -70 to Day 1) | TRTR consists of measuring the degree to which an instrument yield reproducible score at different points in time assessed across a fixed and common time interval for all subjects. TRTR co-relations were based on the two-way mixed effect ICC coefficient estimated using the inter-rater reliability package, version 0.84.1. TRTR estimate of 0.7 and above indicated better retest reliability. TRTR was estimated using mean PROMIS F-SF 7a scores from participants who were stable as defined by a PGI-S-Fatigue change score of zero between screening and baseline. As pre-specified in the SAP, for participants contributing to this outcome measure from INS-415 study, data were collected and analyzed for combined population in which ALIS and ELC groups were pooled. |
| Primary | Response Rate as Assessed by Within-Subject Meaningful Change (WSMC) for QOL-B Respiratory Symptoms Final Score Estimated Via Anchor-Based Methods and Validated Via Empirical Cumulative Distribution Functions (eCDFs) | Baseline to Month 7 | WSMC was estimated via change scores computed between Baseline and end of study (EOS) (Month 7). The estimated WSMC threshold of 14.81 points for the QOL-B Respiratory Symptom score (9-item scale ranging from 0 to 100, higher scores=fewer symptoms and better quality of life) as derived from anchor-based methods supplemented with eCDF curves was used for analysis. The percentage of participants and confidence intervals were estimated by standardized logistic regression with treatment group and history of mycobacterium avium complex (MAC) lung infection as factors in the model. Missing change from baseline at Month 7 was imputed by multiple imputation. The mean of all imputed values was used to derive response according to WSMC. Response rate was expressed in terms of percentage of participants and the percentages are rounded off to the nearest decimal. |
| Primary | Response Rate as Assessed by WSMC for PROMIS Fatigue Final Score Estimated Via Anchor-Based Methods and Validated Via eCDFs | Baseline to Month 7 | WSMC was estimated via change scores computed between Baseline and EOS (Month 7). The percentage of participants and confidence intervals were estimated by standardized logistic regression with treatment group and history of MAC lung infection as factors in the model. Missing change from baseline at Month 7 was imputed by multiple imputation. The mean of all imputed values was used to derive response according to WSMC. The estimated WSMC threshold of -4.00 points for the PROMIS Fatigue score as derived from anchor-based methods supplemented with eCDF curves was used for analysis. Response rate was expressed in terms of percentage of participants and the percentages are rounded off to the nearest decimal. |
| Secondary | Percentage of Participants Achieving Culture Conversion by Month 6 | Baseline to Month 6 | Culture conversion by Month 6 was defined as no MAC growth on agar media and broth media in all sputum cultures at 2 consecutive visits up to Month 6. Percentage of participants and confidence intervals were estimated by standardized logistic regression with treatment group and history of MAC lung infection as factors in the model. For the purpose of the estimation missing conversion status by Month 6 was imputed by multiple imputation. Percentages are rounded off to the nearest decimal. |
| Secondary | Change From Baseline in QOL-B Respiratory Symptom Score at Month 7 | Baseline to Month 7 | The QOL-B is a self-administered, PRO questionnaire used to assess symptoms, functioning, and health related quality of life in adults with lung conditions. The respiratory symptom domain of the QOL-B contains 9 items describing patient's self-assessment of her/his respiratory symptoms that affect daily life. For each of the 8 items (chest congestion, coughing, cough up mucus, shortness of breath with greater activity, wheezing, chest pain, shortness of breath when talking, woken up during night due to cough), scores ranged from 1 to 4 (1= lot, 2= moderate, 3= little, 4= not at all) and the sputum item based on the color ranged from 0=don't know,1=green with traces of blood/brownish dark,2=yellowish-green,3=clear to yellow,4=clear. The item scores were summed and then standardized on a 0 to 100-point scale to derive the domain score with higher scores representing fewer symptoms or better functioning and quality of life. Positive change from baseline indicates improvement. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Psychometric Cross-Sectional Validation of Patient Reported Outcome (PRO): Patient Global Impression of Severity (PGI-S) Respiratory Scale Score at Baseline | Baseline | ITT Analysis Set comprises all participants who were randomized. | score on scale | MEAN |
| PRIMARY | Psychometric Cross-Sectional Validation of PRO: PGI-S Fatigue Scale Score at Baseline | Baseline | ITT Analysis Set comprises all participants who were randomized. | score on scale | MEAN |
| PRIMARY | Psychometric Cross-Sectional Validation of PRO: Quality of Life Questionnaire - Bronchiectasis (QoL-B) Respiratory Symptoms Scale Score at Baseline | Baseline | The ITT Analysis Set comprises all participants who were randomized. | score on scale | MEAN |
| PRIMARY | Psychometric Cross-Sectional Validation of PRO: Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Score at Baseline | Baseline | The ITT Analysis Set comprises all participants who were randomized. | score on scale | MEAN |
| PRIMARY | Assessment of Test-Retest Reliability (TRTR) Reported as the Intraclass Co-relation (ICC) Estimate Among Participants Reporting no Change on Respiratory PGI-S Score Applied to QOL-B Respiratory Domain Score Between Screening and Baseline | From Screening to Baseline (Day -70 to Day 1) | As pre-specified in the SAP, to adequately power the planned modern psychometric methods (MPMs) the cross-sectional validation sample was composed of a total of 97 participants enrolled in the INS-415 study (comprising of pooled ALIS + ELC data per planned analyses) who provided item-level QOL-B Respiratory domain data and Screening/Baseline data from the first 132 participants enrolled in the INS-416 study (NCT04677569) to yield a total sample of n=229. | ICC estimate | NUMBER |
| PRIMARY | Assessment of TRTR Reported as the ICC Estimate Among Participants Reporting no Change on Fatigue PGI-S Score Applied to PROMIS F-SF 7a Score Between Screening and Baseline | From Screening to Baseline (Day -70 to Day 1) | As pre-specified in the SAP, to adequately power the planned MPMs the cross-sectional validation sample was composed of a total of 98 participants enrolled in the INS-415 study (comprising of pooled ALIS + ELC data per planned analyses) who provided item-level PROMIS F-SF 7a data and Screening/Baseline data from the first 132 participants enrolled in the INS-416 study to yield a total sample of n=230. | ICC estimate | NUMBER |
| PRIMARY | Response Rate as Assessed by Within-Subject Meaningful Change (WSMC) for QOL-B Respiratory Symptoms Final Score Estimated Via Anchor-Based Methods and Validated Via Empirical Cumulative Distribution Functions (eCDFs) | Baseline to Month 7 | The ITT Analysis Set comprises all participants who were randomized. | percentage of participants | NUMBER |
| PRIMARY | Response Rate as Assessed by WSMC for PROMIS Fatigue Final Score Estimated Via Anchor-Based Methods and Validated Via eCDFs | Baseline to Month 7 | The ITT Analysis Set comprises all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving Culture Conversion by Month 6 | Baseline to Month 6 | The ITT Analysis Set comprises all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in QOL-B Respiratory Symptom Score at Month 7 | Baseline to Month 7 | The ITT Analysis Set comprises all participants who were randomized. | score on scale | LEAST_SQUARES_MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Psychometric Cross-Sectional Validation of Patient Reported Outcome (PRO): Patient Global Impression of Severity (PGI-S) Respiratory Scale Score at Baseline | ALIS + Background Regimen (Azithromycin + Ethambutol) | 2.5 | Standard Deviation 0.82 | score on scale | |
| Psychometric Cross-Sectional Validation of Patient Reported Outcome (PRO): Patient Global Impression of Severity (PGI-S) Respiratory Scale Score at Baseline | ELC + Background Regimen (Azithromycin + Ethambutol) | 2.4 | Standard Deviation 0.90 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: PGI-S Fatigue Scale Score at Baseline | ALIS + Background Regimen (Azithromycin + Ethambutol) | 2.5 | Standard Deviation 0.99 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: PGI-S Fatigue Scale Score at Baseline | ELC + Background Regimen (Azithromycin + Ethambutol) | 2.5 | Standard Deviation 0.81 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: Quality of Life Questionnaire - Bronchiectasis (QoL-B) Respiratory Symptoms Scale Score at Baseline | ALIS + Background Regimen (Azithromycin + Ethambutol) | 63.04 | Standard Deviation 14.824 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: Quality of Life Questionnaire - Bronchiectasis (QoL-B) Respiratory Symptoms Scale Score at Baseline | ELC + Background Regimen (Azithromycin + Ethambutol) | 66.90 | Standard Deviation 15.673 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Score at Baseline | ALIS + Background Regimen (Azithromycin + Ethambutol) | 18.3 | Standard Deviation 5.76 | score on scale | |
| Psychometric Cross-Sectional Validation of PRO: Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Score at Baseline | ELC + Background Regimen (Azithromycin + Ethambutol) | 18.6 | Standard Deviation 4.76 | score on scale | |
Assessment of Test-Retest Reliability (TRTR) Reported as the Intraclass Co-relation (ICC) Estimate Among Participants Reporting no Change on Respiratory PGI-S Score Applied to QOL-B Respiratory Domain Score Between Screening and Baseline | ALIS or ELC Along With Background Regimen (Azithromycin + Ethambutol): Combined Group | 0.69 | ICC estimate | ||
| Assessment of TRTR Reported as the ICC Estimate Among Participants Reporting no Change on Fatigue PGI-S Score Applied to PROMIS F-SF 7a Score Between Screening and Baseline | ALIS or ELC Along With Background Regimen (Azithromycin + Ethambutol): Combined Group | 0.76 | ICC estimate |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ALIS + Background Regimen (Azithromycin + Ethambutol) | 48 | |
| Overall Study | STARTED | ELC + Background Regimen (Azithromycin + Ethambutol) | 51 | |
| Overall Study | COMPLETED | ALIS + Background Regimen (Azithromycin + Ethambutol) | 44 | |
| Overall Study | COMPLETED | ELC + Background Regimen (Azithromycin + Ethambutol) | 48 | |
| Overall Study | NOT COMPLETED | ALIS + Background Regimen (Azithromycin + Ethambutol) | 4 | |
| Overall Study | NOT COMPLETED | ELC + Background Regimen (Azithromycin + Ethambutol) | 3 |
Source ClinicalTrials.gov
Adverse events82
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 | Tinnitus | Ear and labyrinth disorders | ALIS + Background Regimen (Azithromycin + Ethambutol) | 2 | 48 | |
| other | Tinnitus | Ear and labyrinth disorders | ELC + Background Regimen (Azithromycin + Ethambutol) | 4 | 51 | |
| other | Vision blurred | Eye disorders | ALIS + Background Regimen (Azithromycin + Ethambutol) | 0 | 48 | |
| other | Vision blurred | Eye disorders | ELC + Background Regimen (Azithromycin + Ethambutol) | 3 | 51 | |
| other | Diarrhoea | Gastrointestinal disorders | ALIS + Background Regimen (Azithromycin + Ethambutol) | 13 | 48 | |
| other | Diarrhoea | Gastrointestinal disorders | ELC + Background Regimen (Azithromycin + Ethambutol) | 13 | 51 | |
| other | Nausea | Gastrointestinal disorders | ALIS + Background Regimen (Azithromycin + Ethambutol) | 5 | 48 | |
| other | Nausea | Gastrointestinal disorders | ELC + Background Regimen (Azithromycin + Ethambutol) | 4 | 51 | |
| other | Abdominal pain | Gastrointestinal disorders | ALIS + Background Regimen (Azithromycin + Ethambutol) | 4 | 48 | |
| other | Abdominal pain | Gastrointestinal disorders | ELC + Background Regimen (Azithromycin + Ethambutol) | 4 | 51 |
1 to 10 of 82Source ClinicalTrials.gov
See all 82 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ALIS + Background Regimen (Azithromycin + Ethambutol) | 0 | 48 |
| Total, all-cause mortality | ELC + Background Regimen (Azithromycin + Ethambutol) | 0 | 51 |
| Total, other adverse events | ALIS + Background Regimen (Azithromycin + Ethambutol) | 38 | 48 |
| Total, other adverse events | ELC + Background Regimen (Azithromycin + Ethambutol) | 33 | 51 |
| Total, serious adverse events | ALIS + Background Regimen (Azithromycin + Ethambutol) | 7 | 48 |
| Total, serious adverse events | ELC + Background Regimen (Azithromycin + Ethambutol) | 3 | 51 |
Source ClinicalTrials.gov
Papers2
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| ALIS + Background Regimen (Azithromycin + Ethambutol) vs ELC + Background Regimen (Azithromycin + Ethambutol): Number of Participants Who Experience Any Treatment-emergent Adverse Event (TEAE) | Trial comparisons |
| ALIS + Background Regimen (Azithromycin + Ethambutol) vs ELC + Background Regimen (Azithromycin + Ethambutol): deaths from any cause | Trial comparisons |
| ALIS + Background Regimen (Azithromycin + Ethambutol) vs ELC + Background Regimen (Azithromycin + Ethambutol): 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.
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GET https://conductscience.org/api/trials/v1/NCT04677543/results?page=2