A Phase 3 interventional study of CMS and Placebo in Non Cystic Fibrosis Bronchiectasis, sponsored by Zambon SpA. Terminated at 89 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-15.
Sponsored by Zambon SpA · Phase 3, Interventional, and Prevention
The primary objective of the trial was to investigate the effect of the use of inhaled colistimethate sodium (CMS), administered twice a day (b.i.d.) via a specific nebulizer for 12 months, compared to placebo in subjects with non-cystic fibrosis bronchiectasis (NCFB) chronically infected with P. aeruginosa on the annualised frequency of pulmonary exacerbations.
This was a randomised, multi-centre, double-blind, placebo-controlled, parallel-group interventional trial in subjects with NCFB chronic P. aeruginosa infection. Subjects were randomised to CMS or placebo in a 1:1 ratio. The study consisted of 7 clinic visits with a follow-up phone call 12.5 month after randomisation or 2 weeks after discontinuation of treatment. Additional clinic visits, where feasible, and weekly phone calls were conducted during or after pulmonary exacerbations (or any episodes of pneumonia) until resolution.
Every effort was made to have all planned and unscheduled visits at the study site. Mandatory on-site visits were Screening Visit (Visit 1) and Randomisation (Visit 2). However, if one of the visits after Visit 2 could not be performed at site due to COVID-19, remote visits (e.g., by telephone) were permitted. If the final visit (Visit 7) had to be conducted remotely, the subjects were asked to return to the clinic for on-site assessments at the earliest opportunity.
After consulting with the US Food and Drug Administration, the study was brought to an early close primarily due to the difficulty of recruiting subjects in the context of the COVID pandemic, but also due to the potential for loss of scientific equipoise and the ethical implications of continuing to expose subjects to placebo given the positive results from PROMIS I. Recruitment to PROMIS II was stopped as of 27 October 2021, with the study terminated as of 15 March 2022. The study was not stopped prematurely due to any safety or futility concerns and the accrued data were fully analysed and are presented.
404 studies on the registry are indexed under Bronchiectasis; 125 are open to participants now.
This study's enrollment of 287 is above the median of 60 across 263 interventional studies indexed under Bronchiectasis.
Browse Bronchiectasis studies →Zambon SpA is the lead sponsor of 23 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Inhaled colistimethate sodium twice daily. The active pharmaceutical ingredient consisting of pure CMS one million international units (MIU) / 80 mg of CMS / 33 mg colistin base activity (CBA) was provided as a powder for nebuliser solution in 10R Internation Organization for Standardization (ISO) glass vials.
Drug: CMS
Saline solution inhaled twice daily, provided and administered at the same way of the IMP.
Other: Placebo
1 MIU equivalent to 80 mg colistimethate sodium diluted in 1 mL saline solution 0.45%. Investigational Medicinal Product (IMP) glass vials were shrink wrapped in opaque white plastic and provided in boxes of 30 vials (two weeks of b.i.d. dosing). The 1 MIU/ml CMS/0.45% saline solution was transferred from the glass vial into a specific nebuliser system fitted with a 0.3 mL medication chamber, for administration by inhalation. This delivered a nominal dose of 0.3 MIU/24 mg CMS (11 mg CBA) from the device. The first dose of the IMP was administered at the site under the supervision of the site staff and subjects were instructed how to prepare and self-administer the IMP at home via a specific nebuliser system, b.i.d. (morning and evening) over aperiod of 12 month. At least 10 minutes (min) before each administration, an inhaled short-acting bronchodilator (e.g. salbutamol/albuterol), supplied by the Sponsor, could be taken to improve tolerability.
Also known as: Promixin
1 mL saline solution 0.45%. The placebo was made up of identical empty glass vials to which the same saline solution diluent was added in exactly the same way as the reconstitution of the active treatment by injecting the diluent through the rubber stopper. The glass vials were shrink wrapped with opaque white plastic to mantain the blind.
Also known as: Saline Solution
Mean Annual Non-cystic Fibrosis Bronchiectasis (NCFB) Pulmonary Exacerbation Rate
The primary efficacy assessment for an individual subject was the frequency of pulmonary exacerbations (exacerbation rate). A pulmonary exacerbation was defined as the presence concurrently of at least three of the following eight symptoms/signs for at least 24 hours: * increased cough; * increased sputum volume and/or consistency; * increased sputum purulence; * new or increased haemoptysis; * increased wheezing; * increased dyspnoea; * increased fatigue/malaise and * episodes of fever (temperature ≥38°C). AND It was clinically determined that the subject required and was prescribed systemic antibiotic therapy. AND The episode of exacerbation lasted for at least 24 hours. The overall episode of exacerbation needs to last at least 24 hours, but individual symptoms/signs can last less than 24 hours (e.g, a temperature)
Time frame: 12 months
Time to First NCFB Pulmonary Exacerbation
Time to First NCFB Pulmonary Exacerbation (PE) (days) = start date of first NCFB PE - date of randomization. Subjects who completed or discontinued the trial with no NCFB PE are censored at the date of treatment completion/discontinuation.
Time frame: 12 months
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score
The Saint George's Respiratory Questionnaire (SGRQ) is a validated 50-item symptom questionnaire developed to measure health status and Quality of Life (QoL) in subjects with obstructive airway diseases. The scores range from 0 (best possible health status) to 100 (worst possible health status). The total score is calculated as: (sum of the weights of the positive items of all domains / sum of the weights of all items of all domains)\*100. Change from Baseline was calculated as the SGRQ total score at the visit minus Baseline. Change from baseline in SGRQ total score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
P. Aeruginosa Density: Change From Baseline at Visit 3 and at Visit 7
The P. aeruginosa density as determined by the change in log10 CFU/mL sputum from baseline (Visit 2) to Day 28 (Visit 3) was compared between the treatment groups by an ANCOVA model including treatment, pooled site, i.e. country and use of stable concomitant therapy with oral macrolides as fixed effects and baseline value as covariate. Least square means in each treatment group, least square mean difference between treatments, their 95% CIs and associated p-value were estimated.
Time frame: Visit 3 (28 days) and Visit 7 (12 months) / EoT
P. Aeruginosa Density: Change From Baseline
Summary statistics of the P. aeruginosa density (i.e. quantification, log10 CFU/mL sputum) and absolute change from baseline were provided by treatment group for each trial visit.
Time frame: Up to Visit 7 (12 months) / EoT
Mean Annual Severe Non-cystic Fibrosis Bronchiectasis (NCFB) Pulmonary Exacerbation Rate
A severe pulmonary exacerbation was defined as requiring intravenous antibiotics and/or hospitalisation.
Time frame: 12 months
Time to First Severe NCFB Pulmonary Exacerbation
Time to First severe NCFB Pulmonary Exacerbation (PE) (days) = start date of first severe NCFB PE - date of randomization. Subjects who completed or discontinued the trial with no severe NCFB PE are censored at the date of treatment completion/discontinuation. Severe exacerbations are defined as those requiring IV antibiotics and/or hospitalisation.
Time frame: 12 months
NCFB Pulmonary Exacerbation Free Days
Annualised number of exacerbation-free days = (follow-up time \[days\] - sum of durations of NCFB pulmonary exacerbations \[days\]) / follow-up time (years). For NCFB pulmonary exacerbations with a resolution date after the treatment completion/discontinuation, the number of days of duration after the treatment completion/discontinuation will be subtracted from the sum of durations of NCFB pulmonary exacerbations. Non-parametric ANCOVA model with a randomisation-based methodology was used. The model included treatment group, country, and use of stable concomitant therapy with oral macrolides as covariates.
Time frame: 12 months
Days of Work Absence
Number of Days Work Absence is computed for each subject by summing all days of work absence.
Time frame: 12 months
Days of Work Absence Per Employment Month
Number of Days Work Absence per Employment Month = Number of Days of Work Absence / Overall Employment Time (months). Number of Days Work Absence is computed for each subject by summing all days of work absence.
Time frame: 12 months
Subjects With Hospitalisations Due to NCFB Pulmonary Exacerbations
The number and percentage of subjects with at least one hospitalisation for a NCFB pulmonary exacerbation are provided. Percentages were computed on the total number of subjects with at least one pulmonary exacerbation.
Time frame: 12 months
Mean Annual Hospitalisation for NCFB Pulmonary Exacerbation Rate
The annual rate of hospitalisation for NCFB pulmonary exacerbation
Time frame: 12 months
Change From Baseline in QoL-B Physical Functioning Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Physical Functioning Domain, the items 1, 2, 3, 4, 16 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Role Functioning Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Role Functioning Domain, the items 17, 20, 25, 27, 28 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Vitality Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Vitality Domain the items 6, 8, 9 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Emotional Functioning Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Emotional Functioning Domain the items 7, 10, 11, 23 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Social Functioning Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Social Functioning Domain the items 18, 19, 22, 26 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Treatment Burden Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Treatment Burden Domain the items 12, 13, 14 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Health Perceptions Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Health Perceptions Domain the items 5, 15, 21, 24 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Change From Baseline in QoL-B Respiratory Symptoms Domain Score
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Respiratory Symptoms Domain the items from 29 to 37 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
Time frame: Up to Visit 7 (12 months) / EoT
Recruitment was halted with 287 subjects randomised.
| Milestone | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Started | 152 | 135 |
| Modified intention to treat population - (mitt) | 152 | 135 |
| Safety population - (saf) | 152 | 135 |
| Per-protocol population - (pp) | 112 | 98 |
| Completed | 94 | 89 |
| Not completed | 58 | 46 |
| Withdrew: Withdrawal by subject | 11 | 14 |
| Withdrew: Study terminated by the sponsor | 29 | 19 |
| Withdrew: Adverse event | 10 | 5 |
| Withdrew: Death | 3 | 2 |
| Withdrew: Non-compliance with study drug | 3 | 0 |
| Withdrew: Protocol-specified withdrawal criterion met | 0 | 5 |
| Withdrew: Other | 2 | 1 |
The primary efficacy assessment for an individual subject was the frequency of pulmonary exacerbations (exacerbation rate). A pulmonary exacerbation was defined as the presence concurrently of at least three of the following eight symptoms/signs for at least 24 hours: * increased cough; * increased sputum volume and/or consistency; * increased sputum purulence; * new or increased haemoptysis; * increased wheezing; * increased dyspnoea; * increased fatigue/malaise and * episodes of fever (temperature ≥38°C). AND It was clinically determined that the subject required and was prescribed systemic antibiotic therapy. AND The episode of exacerbation lasted for at least 24 hours. The overall episode of exacerbation needs to last at least 24 hours, but individual symptoms/signs can last less than 24 hours (e.g, a temperature)
| events/year | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Mean Annual Non-cystic Fibrosis Bronchiectasis (NCFB) Pulmonary Exacerbation Rate | 0.889 (0.722 to 1.093) | 0.885 (0.710 to 1.103) |
Time to First NCFB Pulmonary Exacerbation (PE) (days) = start date of first NCFB PE - date of randomization. Subjects who completed or discontinued the trial with no NCFB PE are censored at the date of treatment completion/discontinuation.
| days | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Time to First NCFB Pulmonary Exacerbation | 267.0 (189.0 to 357.0) | 214.0 (177.0 to 279.0) |
The Saint George's Respiratory Questionnaire (SGRQ) is a validated 50-item symptom questionnaire developed to measure health status and Quality of Life (QoL) in subjects with obstructive airway diseases. The scores range from 0 (best possible health status) to 100 (worst possible health status). The total score is calculated as: (sum of the weights of the positive items of all domains / sum of the weights of all items of all domains)\*100. Change from Baseline was calculated as the SGRQ total score at the visit minus Baseline. Change from baseline in SGRQ total score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | -1.758 (-4.299 to 0.783) | -4.502 (-7.213 to -1.790) |
| Visit 4 (3 months) | -3.901 (-6.520 to -1.281) | -4.603 (-7.375 to -1.831) |
| Visit 5 (6 months) | -4.598 (-7.325 to -1.871) | -5.518 (-8.359 to -2.677) |
| Visit 6 (9 months) | -2.953 (-6.302 to 0.396) | -4.544 (-7.989 to -1.100) |
| Visit 7 (12 months) / EoT | -5.044 (-8.463 to -1.625) | -3.200 (-6.984 to 0.584) |
The P. aeruginosa density as determined by the change in log10 CFU/mL sputum from baseline (Visit 2) to Day 28 (Visit 3) was compared between the treatment groups by an ANCOVA model including treatment, pooled site, i.e. country and use of stable concomitant therapy with oral macrolides as fixed effects and baseline value as covariate. Least square means in each treatment group, least square mean difference between treatments, their 95% CIs and associated p-value were estimated.
| log10 CFU/mL | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | -1.679 (-1.975 to -1.383) | -0.081 (-0.365 to 0.203) |
| Visit 7 (12 months) / EoT | -0.639 (-1.121 to -0.156) | 0.074 (-0.396 to 0.545) |
Summary statistics of the P. aeruginosa density (i.e. quantification, log10 CFU/mL sputum) and absolute change from baseline were provided by treatment group for each trial visit.
| log10 CFU/mL | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | -1.79 ± 1.616 | 0.02 ± 1.312 |
| Visit 5 (6 months) | -1.33 ± 1.501 | 0.13 ± 1.487 |
| Visit 7 (12 months) / EoT | -0.62 ± 1.600 | -0.06 ± 1.376 |
A severe pulmonary exacerbation was defined as requiring intravenous antibiotics and/or hospitalisation.
| events/year | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Mean Annual Severe Non-cystic Fibrosis Bronchiectasis (NCFB) Pulmonary Exacerbation Rate | 0.161 (0.101 to 0.256) | 0.121 (0.070 to 0.209) |
Time to First severe NCFB Pulmonary Exacerbation (PE) (days) = start date of first severe NCFB PE - date of randomization. Subjects who completed or discontinued the trial with no severe NCFB PE are censored at the date of treatment completion/discontinuation. Severe exacerbations are defined as those requiring IV antibiotics and/or hospitalisation.
| days | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Time to First Severe NCFB Pulmonary Exacerbation | NA (NA to NA) | NA (NA to NA) |
Annualised number of exacerbation-free days = (follow-up time \[days\] - sum of durations of NCFB pulmonary exacerbations \[days\]) / follow-up time (years). For NCFB pulmonary exacerbations with a resolution date after the treatment completion/discontinuation, the number of days of duration after the treatment completion/discontinuation will be subtracted from the sum of durations of NCFB pulmonary exacerbations. Non-parametric ANCOVA model with a randomisation-based methodology was used. The model included treatment group, country, and use of stable concomitant therapy with oral macrolides as covariates.
| days | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| NCFB Pulmonary Exacerbation Free Days | 342.592 (336.871 to 348.313) | 344.683 (338.611 to 350.756) |
Number of Days Work Absence is computed for each subject by summing all days of work absence.
| days | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Days of Work Absence | 1.0 ± 6.40 | 0.5 ± 2.70 |
Number of Days Work Absence per Employment Month = Number of Days of Work Absence / Overall Employment Time (months). Number of Days Work Absence is computed for each subject by summing all days of work absence.
| days per month | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Days of Work Absence Per Employment Month | 0.3 ± 1.70 | 0.1 ± 0.36 |
The number and percentage of subjects with at least one hospitalisation for a NCFB pulmonary exacerbation are provided. Percentages were computed on the total number of subjects with at least one pulmonary exacerbation.
| Participants | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Subjects with NCFB Pulmonary Exacerbation | 72 | 64 |
| Subjects with >=1 Hospitalisation for NCFB Pulmonary Exacerbation | 12 | 10 |
The annual rate of hospitalisation for NCFB pulmonary exacerbation
| events/year | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Mean Annual Hospitalisation for NCFB Pulmonary Exacerbation Rate | 0.107 (0.063 to 0.182) | 0.097 (0.054 to 0.175) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Physical Functioning Domain, the items 1, 2, 3, 4, 16 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 2.082 (-1.522 to 5.686) | 4.457 (0.601 to 8.313) |
| Visit 4 (3 months) | 1.985 (-1.987 to 5.957) | 2.756 (-1.448 to 6.960) |
| Visit 5 (6 months) | 4.918 (0.493 to 9.343) | 1.764 (-2.802 to 6.331) |
| Visit 6 (9 months) | 3.583 (-1.198 to 8.365) | 3.135 (-1.816 to 8.086) |
| Visit 7 (12 months) / EoT | 2.006 (-2.137 to 6.148) | 0.818 (-3.503 to 5.140) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Role Functioning Domain, the items 17, 20, 25, 27, 28 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 2.178 (-1.086 to 5.442) | 5.953 (2.471 to 9.435) |
| Visit 4 (3 months) | 3.299 (-0.179 to 6.776) | 5.931 (2.261 to 9.601) |
| Visit 5 (6 months) | 4.104 (0.302 to 7.906) | 6.657 (2.725 to 10.590) |
| Visit 6 (9 months) | 1.464 (-2.694 to 5.623) | 3.338 (-0.940 to 7.615) |
| Visit 7 (12 months) / EoT | 3.131 (-0.550 to 6.811) | 4.310 (0.485 to 8.134) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Vitality Domain the items 6, 8, 9 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 1.601 (-1.775 to 4.977) | 1.522 (-2.093 to 5.137) |
| Visit 4 (3 months) | 2.449 (-0.902 to 5.799) | 2.142 (-1.400 to 5.684) |
| Visit 5 (6 months) | 3.832 (-0.323 to 7.987) | 1.614 (-2.664 to 5.892) |
| Visit 6 (9 months) | 2.567 (-1.944 to 7.078) | -0.290 (-4.920 to 4.340) |
| Visit 7 (12 months) / EoT | 4.081 (0.601 to 7.561) | 3.282 (-0.331 to 6.895) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Emotional Functioning Domain the items 7, 10, 11, 23 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 2.582 (-0.265 to 5.428) | 1.245 (-1.819 to 4.308) |
| Visit 4 (3 months) | 2.810 (-0.213 to 5.833) | 2.564 (-0.651 to 5.778) |
| Visit 5 (6 months) | 2.023 (-1.286 to 5.331) | 3.123 (-0.269 to 6.515) |
| Visit 6 (9 months) | 0.981 (-3.103 to 5.065) | -0.409 (-4.584 to 3.766) |
| Visit 7 (12 months) / EoT | 2.795 (-0.207 to 5.797) | 3.880 (0.754 to 7.005) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Social Functioning Domain the items 18, 19, 22, 26 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 4.265 (0.312 to 8.218) | 3.686 (-0.549 to 7.921) |
| Visit 4 (3 months) | 5.287 (1.013 to 9.561) | 5.474 (0.932 to 10.016) |
| Visit 5 (6 months) | 4.285 (-0.395 to 8.964) | 1.438 (-3.436 to 6.313) |
| Visit 6 (9 months) | 3.485 (-1.440 to 8.410) | 1.541 (-3.527 to 6.609) |
| Visit 7 (12 months) / EoT | 3.118 (-0.973 to 7.209) | -0.551 (-4.804 to 3.702) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Treatment Burden Domain the items 12, 13, 14 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | -2.508 (-6.319 to 1.303) | -3.006 (-7.219 to 1.207) |
| Visit 4 (3 months) | -3.609 (-7.599 to 0.381) | -2.236 (-6.533 to 2.061) |
| Visit 5 (6 months) | 1.098 (-3.446 to 5.641) | 0.158 (-4.367 to 4.684) |
| Visit 6 (9 months) | -0.544 (-5.324 to 4.236) | -7.371 (-12.418 to -2.324) |
| Visit 7 (12 months) / EoT | -1.041 (-5.397 to 3.316) | -2.363 (-6.919 to 2.193) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Health Perceptions Domain the items 5, 15, 21, 24 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 4.746 (1.695 to 7.798) | 4.819 (1.544 to 8.094) |
| Visit 4 (3 months) | 4.905 (1.578 to 8.232) | 5.143 (1.609 to 8.678) |
| Visit 5 (6 months) | 8.185 (4.548 to 11.822) | 5.658 (1.908 to 9.408) |
| Visit 6 (9 months) | 6.248 (2.470 to 10.026) | 5.959 (2.086 to 9.832) |
| Visit 7 (12 months) / EoT | 5.474 (2.222 to 8.726) | 4.042 (0.658 to 7.426) |
The Quality of Life Bronchiectasis (QoL-B) is a validated self-administered, patient reported outcome measure assessing symptoms, functioning and health-related QoL for subjects with NCFB. It contains 37 items in 8 scales (Respiratory Symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each QoL-B domain is assessed on a scale of 0 to 100, higher scores indicate better health status. For the Respiratory Symptoms Domain the items from 29 to 37 are considered. Change from baseline in QoL-B domain score at Visit 3, Visit 4, Visit 5, Visit 6 and Visit 7 were analysed using a linear mixed model for repeated measures including treatment, visit, treatment-by-visit interaction, use of stable concomitant therapy with oral macrolides and pooled site, i.e. country, as fixed effects and baseline value as covariate. An unstructured covariance matrix was assumed and the Kenward-Roger adjustment was used for the degrees of freedom.
| scores on a scale | CMS (Colistimethate Sodium) | Placebo |
|---|---|---|
| Visit 3 (28 days) | 5.133 (2.532 to 7.734) | 4.013 (1.224 to 6.802) |
| Visit 4 (3 months) | 4.223 (1.328 to 7.118) | 5.532 (2.455 to 8.609) |
| Visit 5 (6 months) | 5.960 (2.811 to 9.109) | 4.750 (1.505 to 7.995) |
| Visit 6 (9 months) | 6.982 (3.197 to 10.767) | 4.586 (0.708 to 8.463) |
| Visit 7 (12 months) / EoT | 4.947 (2.172 to 7.722) | 4.594 (1.707 to 7.482) |
Collected over All AEs occurring from Visit 2 (day 0 = first IMP administration) until the follow-up phone call (2 weeks +/- 3 days after the end of treatment = totally up to 54 weeks +/- 3 days from the study Day 0.. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CMS (Colistimethate Sodium) (SAF) | 3/152 (2%) | 27/152 (17.8%) | 123/152 (80.9%) |
| Placebo (SAF) | 2/135 (1.5%) | 17/135 (12.6%) | 104/135 (77%) |
| Event | CMS (Colistimethate Sodium) (SAF) | Placebo (SAF) |
|---|---|---|
| Infective exacerbation of bronchiectasisInfections and infestations | 12/152 | 10/135 |
| COVID-19Infections and infestations | 3/152 | 0/135 |
| AsthmaRespiratory, thoracic and mediastinal disorders | 2/152 | 0/135 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 2/152 | 0/135 |
| PneumoniaInfections and infestations | 1/152 | 1/135 |
| Pseudomonas infectionInfections and infestations | 0/152 | 1/135 |
| Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders | 0/152 | 1/135 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 0/152 | 1/135 |
| Head injuryInjury, poisoning and procedural complications | 0/152 | 1/135 |
| Wrist fractureInjury, poisoning and procedural complications | 0/152 | 1/135 |
| Event | CMS (Colistimethate Sodium) (SAF) | Placebo (SAF) |
|---|---|---|
| Infective exacerbation of bronchiectasisInfections and infestations | 69/152 | 67/135 |
| CoughRespiratory, thoracic and mediastinal disorders | 11/152 | 9/135 |
| HeadacheNervous system disorders | 7/152 | 8/135 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 9/152 | 5/135 |
| COVID-19Infections and infestations | 8/152 | 4/135 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 7/152 | 6/135 |
| BronchospasmRespiratory, thoracic and mediastinal disorders | 7/152 | 3/135 |
| SinusitisInfections and infestations | 6/152 | 6/135 |
| DiarrhoeaGastrointestinal disorders | 5/152 | 6/135 |
| NasopharyngitisInfections and infestations | 2/152 | 6/135 |
The modified Intention-To-Treat (mITT) Population is the Full Analysis Set and comprised all subjects who provided informed consent, were randomised and received at least 1 dose or partial dose of the IMP.
| Age, Categorical(Participants) | CMS (Colistimethate Sodium) | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 85 | 73 | 158 |
| >=65 years | 67 | 62 | 129 |
| Age, Continuous(years) | CMS (Colistimethate Sodium) | Placebo | Total |
|---|---|---|---|
| Mean | 59.9 ± 15.19 | 59.6 ± 14.73 | 59.7 ± 14.95 |
| Sex: Female, Male(Participants) | CMS (Colistimethate Sodium) | Placebo | Total |
|---|---|---|---|
| Female | 104 | 94 | 198 |
| Male | 48 | 41 | 89 |
| Race (NIH/OMB)(Participants) | CMS (Colistimethate Sodium) | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 2 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 3 | 3 | 6 |
| White | 146 | 130 | 276 |
| More than one race | 1 | 1 | 2 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Region of Enrollment(participants) | CMS (Colistimethate Sodium) | Placebo | Total |
|---|---|---|---|
| Greece | 2 | 1 | 3 |
| New Zealand | 4 | 2 | 6 |
| Canada | 9 | 10 | 19 |
| Argentina | 56 | 52 | 108 |
| United States | 30 | 20 | 50 |
| Poland | 22 | 26 | 48 |
| Italy | 1 | 0 | 1 |
| Israel | 1 | 3 | 4 |
| Australia | 7 | 6 | 13 |
| France | 18 | 12 | 30 |
| Portugal | 1 | 1 | 2 |
| Germany | 1 | 2 | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2018 to primary completion in Mar 2022, 49 months.
Results were first posted in Dec 2023, 1 year 9 months after primary completion.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.