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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-003374-14 | EudraCT Number |
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The trial is an open-label, non-controlled, multicenter, pilot clinical trial of inhaled molgramostim (recombinant human granulocyte-macrophage colony stimulating factor; rhGM-CSF) in subjects with persistent pulmonary non-tuberculous mycobacterial (NTM) infection. Participants will be treated for 24-weeks with inhaled molgramostim and followed up for 12-weeks after end of treatment. The primary aim of the trial is to investigate the efficacy of inhaled molgramostim on NTM sputum culture conversion to negative.
The study will comprise a Screening Visit, a Baseline Visit, a 24-week treatment period and a 12-week follow-up period. 30 adult participants with a history of chronic NTM infection with at least 2 positive cultures in the prior 2 years, of which at least one is within the last 6 months prior to Screening, will be considered for enrollment. Participants should provide a positive NTM sputum culture at Screening to be eligible.
Two subgroups of participants will be recruited:
The treatment period will consist of 14 trial visits (Screening [within 10 weeks of Baseline], Baseline, and every 4 weeks to Week 48 [visits at Weeks 28, 36 and 44 included telephone contact, others included clinic visits]) and a Follow-up visit 12 weeks after the end of treatment.
At the Baseline visit, eligible participants will start treatment with molgramostim nebulizer solution, 300 μg, administered by inhalation using an eFlow Nebulizer System. At each visit, sputum samples will be collected for staining and microscopy, and microbiological culture. In addition, clinical assessments including body weight, patient reported outcomes, and diffusion capacity of the lung for carbon monoxide (DLCO) will be conducted at each clinic visit. Spirometry will be assessed at Baseline, and at Weeks 12, 24, 32, 40 and 48. A 6-minute walk test (6-MWT) will be conducted at Baseline, at Weeks 12, 24, 48 and at the 12-week Follow-up visit. Safety laboratory samples will be collected at Screening, Baseline and at Weeks 4, 12, 24, 32, 40, 48 and at the 12-week Follow-up visit. Anti-GM-CSF antibodies will be assessed at Baseline, at Week 4, 12, 24, 32, 48, and at the 12-week Follow-up visit.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Inhaled molgramostim/antimycobacterials | Experimental | Inhaled molgramostim administered in participants who remain sputum culture positive while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit |
|
| Inhaled molgramostim | Experimental | Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug NTM guideline-based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Inhaled molgramostim | Drug | 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Sputum Culture Conversion to Negative | Sputum culture conversion is defined as at least 3 consecutive sputum samples without growth of NTM during the treatment period. | 48 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Sputum Smear Conversion to Negative | Sputum smear conversion is defined as at least three consecutive negative acid-fast bacilli (AFB) stained sputum smears on microscopy during the treatment period among participants who were smear positive at Baseline. | 48 weeks |
| Number of Participants With Durable Sputum Culture Conversion |
Not provided
Inclusion Criteria:
History of chronic pulmonary infection with MAC or M. abscessus (defined as at least 2 documented positive sputum cultures in the prior 2 years, of which at least one was obtained in the 6 months prior to Screening).
Subject fulfills one of the following criteria:
Ability to produce at least 2 mL of sputum or be willing to undergo an induction that produces at least 2 mL of sputum for clinical evaluation.
Female or male ≥18 years of age.
Females who have been post-menopausal for more than 1 year or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with less than 1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone- releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence), during and until thirty (30) days after last dose of trial treatment. Females of childbearing potential must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test at dosing at Baseline (Visit 2) and must not be lactating.
Males agreeing to use condoms during and until thirty (30) days after last dose of medication, or males having a female partner who is using adequate contraception as described above.
Willing and able to provide signed informed consent.
Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the investigator
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Grant Waterer, Prof. | Royal Perth Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Concord Repatriation General Hospital | Concord | New South Wales | 2139 | Australia | ||
| The Prince Charles Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37948736 | Derived | Thomson RM, Loebinger MR, Burke AJ, Morgan LC, Waterer GW, Ganslandt C. OPTIMA: An Open-Label, Noncomparative Pilot Trial of Inhaled Molgramostim in Pulmonary Nontuberculous Mycobacterial Infection. Ann Am Thorac Soc. 2024 Apr;21(4):568-576. doi: 10.1513/AnnalsATS.202306-532OC. |
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Adults with chronic pulmonary NTM infection and a positive sputum culture at Screening were recruited into 2 groups:
This was a multicenter study conducted in a total of 5 sites in Australia and the UK. First participant was enrolled on 1 March 2018 and last participant completed the study on 13 January 2020.
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| ID | Title | Description |
|---|---|---|
| FG000 | Inhaled Molgramostim/Antimycobacterials | Inhaled molgramostim administered in participants who remain sputum culture positive while currently on a multidrug NTM-guideline based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
| FG001 | Inhaled Molgramostim | Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug NTM-guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
No "analysis sets" were defined. All participants were included in the analyses/data presentations, equivalent to a Full Analysis Set.
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| ID | Title | Description |
|---|---|---|
| BG000 | Inhaled Molgramostim/Antimycobacterials | Inhaled molgramostim administered in participants who remain sputum culture positive while currently on a multidrug NTM-guideline based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 With Sputum Culture Conversion to Negative | Sputum culture conversion is defined as at least 3 consecutive sputum samples without growth of NTM during the treatment period. | Posted | Count of Participants | Participants | 48 weeks |
|
Baseline to 12-week follow-up (60 weeks in total).
All trial subjects were carefully monitored for the occurrence of AEs during the trial period from Baseline (Visit 2) to the 12-week Follow-up visit (Visit 15). AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns.
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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 | Inhaled Molgramostim/Antimycobacterials | Inhaled molgramostim administered in participants who remain sputum culture positive while currently on a multidrug NTM-guideline based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypotension | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypotension | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
This was an exploratory study without hypothesis testing. Only descriptive statistics were used.
The study included participants with treatment refractory NTM infection, who were severely affected by their background disease (in most cases bronchiectasis). These participants probably had an even lower potential for improvement than was assumed prior to the study.
The study was further limited by the lack of a placebo control.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Paymond D Pratt, Chief Medical Officer | Savara Inc | +1 512 784 8757 | ray.pratt@savarapharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 4, 2018 | Jan 11, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 19, 2020 | Jan 11, 2023 | SAP_001.pdf |
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| 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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Open-label, non-controlled, multicenter, pilot clinical trial
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|
| Antimycobacterial regimen | Drug | Multidrug NTM guideline-based antimycobacterial regimen |
|
Durability is defined as sputum culture conversion at or before Week 24 and culture still negative for growth of NTM at 12-weeks follow-up. |
| 60 weeks |
| Number of Participants With Durable Sputum Smear Conversion | Durability is defined as sputum smear conversion at or before Week 48 and AFB stained smear still negative for NTM at 12-weeks follow-up among participants who were smear positive at Baseline. | 60 weeks |
| Change From Baseline in Symptom Scores (Assessed Using Lower Respiratory Tract Infections - Visual Analogue Scale) | For each of the clinical symptoms of Lower Respiratory Tract Infections (dyspnea, fatigue, cough, pain, and sputum), the participant assessed the severity using a 10 cm visual analogue scale (VAS) ranging from 0 = no symptoms to 10 = worst possible symptoms. A total LRTI score was calculated by summing up the score of each symptom (i.e., total LRTI score ranged from 0 to 50). | Baseline to Week 48 |
| Change From Baseline in Symptom Scores (Assessed Using Quality of Life Questionnaire - Bronchiectasis (QOL-B)) | The QOL-B questionnaire including 37 items on 8 scales (emotional functioning, health perceptions, physical functioning, respiratory symptoms, role functioning, social functioning, treatment burden, vitality) was used to assess participant's quality of life (QoL). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardised on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and QoL. Scales contain between 3 and 9 items, thus changing 1 answer category will correspond to a change of 11.1 to 3.7 points. | Baseline to Week 48 |
| Change From Baseline in Global Rating of Health (GRH) | GRH was assessed as an interviewer questionnaire, which assesses global health as "excellent, good, fair or poor". For analysis, these were scored as 1 (poor) to 4 (excellent). | Baseline to Week 48 |
| Change From Baseline in Body Weight | Baseline to Week 48 |
| Change From Baseline in 6-Minute Walking Distance (6MWD) | The 6-minute walk test (6MWT) was performed in accordance with the European Respiratory Society/American Thoracic Society (ERS/ATS) guideline by technicians with documented training and experience. The 6MWT was performed twice at each visit. | Baseline to Week 48 |
| Change From Baseline in Dyspnea Scores During a 6MWT | Baseline to Week 48 |
| Number of Adverse Events (AEs) During the Trial Period | All trial subjects were carefully monitored for the occurrence of AEs during the trial period from Baseline (Visit 2) to the 12-week Follow-up visit (Visit 15). AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. | 60 weeks |
| Number of Serious AEs (SAEs) During the Trial Period | SAEs were defined as any untoward medicinal occurrence or effect that at any dose:
| 60 weeks |
| Number of Adverse Drug Reactions (ADRs) During the Trial Period | All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs. | 60 weeks |
| Number of Severe AEs During the Trial Period | All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards. | 60 weeks |
| Number of Participants Withdrawn From Treatment Due to an AE During the Trial Period | 60 weeks |
| Change From Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) | Baseline to Week 48 |
| Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) (% Predicted) | Baseline to Week 48 |
| Change From Baseline in Forced Vital Capacity (FVC) (% Predicted) | Baseline to Week 48 |
| Number of Subjects With Development of Anti-GM-CSF Antibodies in Serum | Analyses for anti-GM-CSF antibodies were performed at a central laboratory. | 60 weeks |
| Chermside West |
| Queensland |
| 4032 |
| Australia |
| Greenslopes Private Hospital | Greenslopes | Queensland | 4120 | Australia |
| Royal Perth Hospital | Perth | Western Australia | 6000 | Australia |
| Royal Brompton Hospital | London | SW3 6NP | United Kingdom |
| Withdrawal by Subject |
|
| BG001 |
| Inhaled Molgramostim |
Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug NTM-guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| NTM type | Participants may have met one or more criteria | Count of Participants | Participants |
|
| Age at NTM diagnosis | Mean | Standard Deviation | years |
|
| Smoking status | Count of Participants | Participants |
|
| Baseline Lower Respiratory Tract Infections - Visual Analogue Scale (LRTI-VAS) Scores | For each of the clinical symptoms of Lower Respiratory Tract Infections (LRTI; dyspnea, fatigue, cough, pain, and sputum), the participant assessed the severity using a 10 cm visual analogue scale (VAS) ranging from 0 = no symptoms to 10 = worst possible symptoms. A total LRTI score was calculated by summing up the score of each symptom (i.e., total LRTI score ranged from 0 to 50). | Mean | Standard Deviation | score on a scale |
|
| Baseline Quality of Life Questionnaire - Bronchiectasis (QOL-B) scale scores | The QOL-B questionnaire including 37 items on 8 scales (emotional functioning, health perceptions, physical functioning, respiratory symptoms, role functioning, social functioning, treatment burden, vitality) was used to assess particpant's quality of life (QoL). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardised on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and QoL. Scales contain between 3 and 9 items, thus changing 1 answer category will correspond to a change of 11.1 to 3.7 points. | Mean | Standard Deviation | score on a scale |
|
| Baseline Global Rating of Health (GRH) scores | Global Rating of Health was assessed as an interviewer questionnaire, which assesses global health as "excellent, good, fair or poor". For analysis, these were scored as 1 (poor) to 4 (excellent). | Mean | Standard Deviation | score on a scale |
|
| Body weight | Mean | Standard Deviation | kilogram |
|
| Baseline 6-Minute Walking Distance (6MWD) | The 6-minute walk test (6MWT) was performed in accordance with the European Respiratory Society/American Thoracic Society (ERS/ATS) guideline by technicians with documented training and experience. The 6MWT was performed twice at each visit. | Mean | Standard Deviation | meters |
|
| Baseline dyspnea scores | Participants scored the severity of dyspnea during a 6MWT using the Borg CR10 Scale. This was done before and immediately after each 6MWT. The post-walk score is reported below. Zero (0) on the scale corresponds to "nothing at all" and 10 to "extremely strong". | Mean | Standard Deviation | score on a scale |
|
| Baseline Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) | DLCO was assessed in accordance with the ERS/ATS task force standards using local, appropriately calibrated equipment. Parameters to be recorded are DLCO (absolute measured) and DLCO (predicted). The percent predicted DLCO is presented below. Two acceptable tests of DLCO were to be conducted and the average of these was used. | Mean | Standard Deviation | percent predicted |
|
| Baseline Forced Expiratory Volume in 1 Second (FEV1) | FEV1 was assessed in accordance with the ERS/ATS task force standards using local, appropriately calibrated equipment. Three valid measurements were made, and the highest value was used. | Mean | Standard Deviation | percent predicted |
|
| Baseline Forced Vital Capacity (FVC) | FVC was assessed in accordance with the ERS/ATS task force standards using local, appropriately calibrated equipment. Three valid measurements were made, and the highest value was used. | Mean | Standard Deviation | percent predicted |
|
Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug NTM-guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation |
|
|
| Secondary | Number of Participants With Sputum Smear Conversion to Negative | Sputum smear conversion is defined as at least three consecutive negative acid-fast bacilli (AFB) stained sputum smears on microscopy during the treatment period among participants who were smear positive at Baseline. | Only participants who were smear positive at Baseline are included in the analysis. | Posted | Count of Participants | Participants | 48 weeks |
|
|
|
| Secondary | Number of Participants With Durable Sputum Culture Conversion | Durability is defined as sputum culture conversion at or before Week 24 and culture still negative for growth of NTM at 12-weeks follow-up. | Posted | Count of Participants | Participants | 60 weeks |
|
|
|
| Secondary | Number of Participants With Durable Sputum Smear Conversion | Durability is defined as sputum smear conversion at or before Week 48 and AFB stained smear still negative for NTM at 12-weeks follow-up among participants who were smear positive at Baseline. | Posted | Count of Participants | Participants | 60 weeks |
|
|
|
| Secondary | Change From Baseline in Symptom Scores (Assessed Using Lower Respiratory Tract Infections - Visual Analogue Scale) | For each of the clinical symptoms of Lower Respiratory Tract Infections (dyspnea, fatigue, cough, pain, and sputum), the participant assessed the severity using a 10 cm visual analogue scale (VAS) ranging from 0 = no symptoms to 10 = worst possible symptoms. A total LRTI score was calculated by summing up the score of each symptom (i.e., total LRTI score ranged from 0 to 50). | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Symptom Scores (Assessed Using Quality of Life Questionnaire - Bronchiectasis (QOL-B)) | The QOL-B questionnaire including 37 items on 8 scales (emotional functioning, health perceptions, physical functioning, respiratory symptoms, role functioning, social functioning, treatment burden, vitality) was used to assess participant's quality of life (QoL). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardised on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and QoL. Scales contain between 3 and 9 items, thus changing 1 answer category will correspond to a change of 11.1 to 3.7 points. | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Global Rating of Health (GRH) | GRH was assessed as an interviewer questionnaire, which assesses global health as "excellent, good, fair or poor". For analysis, these were scored as 1 (poor) to 4 (excellent). | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Body Weight | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | kilogram | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in 6-Minute Walking Distance (6MWD) | The 6-minute walk test (6MWT) was performed in accordance with the European Respiratory Society/American Thoracic Society (ERS/ATS) guideline by technicians with documented training and experience. The 6MWT was performed twice at each visit. | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | meter | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Dyspnea Scores During a 6MWT | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline to Week 48 |
|
|
|
| Secondary | Number of Adverse Events (AEs) During the Trial Period | All trial subjects were carefully monitored for the occurrence of AEs during the trial period from Baseline (Visit 2) to the 12-week Follow-up visit (Visit 15). AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. | Posted | Number | events | 60 weeks |
|
|
|
| Secondary | Number of Serious AEs (SAEs) During the Trial Period | SAEs were defined as any untoward medicinal occurrence or effect that at any dose:
| Posted | Number | events | 60 weeks |
|
|
|
| Secondary | Number of Adverse Drug Reactions (ADRs) During the Trial Period | All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs. | Posted | Number | events | 60 weeks |
|
|
|
| Secondary | Number of Severe AEs During the Trial Period | All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards. | Posted | Number | events | 60 weeks |
|
|
|
| Secondary | Number of Participants Withdrawn From Treatment Due to an AE During the Trial Period | Posted | Count of Participants | Participants | 60 weeks |
|
|
|
| Secondary | Change From Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | percent predicted | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) (% Predicted) | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | percent predicted | Baseline to Week 48 |
|
|
|
| Secondary | Change From Baseline in Forced Vital Capacity (FVC) (% Predicted) | Evaluable participants at Week 48 included in the analysis. | Posted | Mean | Standard Deviation | percent predicted | Baseline to Week 48 |
|
|
|
| Secondary | Number of Subjects With Development of Anti-GM-CSF Antibodies in Serum | Analyses for anti-GM-CSF antibodies were performed at a central laboratory. | Posted | Count of Participants | Participants | 60 weeks |
|
|
|
| 2 |
| 14 |
| 8 |
| 14 |
| 14 |
| 14 |
| EG001 | Inhaled Molgramostim | Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug NTM-guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment Inhaled molgramostim: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation | 1 | 18 | 6 | 18 | 18 | 18 |
| Right ventricular failure | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | MedDRA 21.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Infective exacerbation of chronic obstructive airways disease | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Lung infection pseudomonal | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Mycobacterium avium complex infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
|
| Overdose | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
|
| Weight decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Malaise | General disorders | MedDRA 21.0 | Systematic Assessment |
|
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
|
| Eosinophil count increased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| C-reactive protein increased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Weight decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Blood urine present | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Haemoglobin decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | MedDRA 21.0 | Systematic Assessment |
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| Tinnitus | Ear and labyrinth disorders | MedDRA 21.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Leukocyturia | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Mycobacterium avium complex infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
Not provided
Not provided
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
| Tiredness |
|
| Cough |
|
| Color of phlegm |
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| Pain |
|
| Physical functioning |
|
| Respiratory symptoms |
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| Role functioning |
|
| Social functioning |
|
| Treatment burden |
|
| Vitality |
|