Trial Outcomes & Findings for Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial Infection (NCT NCT03597347)
NCT ID: NCT03597347
Last Updated: 2023-01-10
Results Overview
NTM sputum culture conversion to negative (defined as at least three consecutive negative mycobacterial cultures collected at least 4 weeks apart during the treatment period).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
48 weeks
Results posted on
2023-01-10
Participant Flow
6 sites in US participated in the study. The first participant was enrolled on 20 June 2019 and last participant completed the study on 2 October 2020, the same date the study was terminated.
A total of 28 subjects were screened for the trial. Fourteen subjects were screening failures.
Participant milestones
| Measure |
Not Consistently NTM Sputum Negative
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
NTM Sputum Positive
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Did Not Meet Recommendations for Treatment With a Multidrug NTM Antimycobacterial Treatment
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Overall Study
STARTED
|
7
|
3
|
4
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
7
|
3
|
4
|
Reasons for withdrawal
| Measure |
Not Consistently NTM Sputum Negative
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
NTM Sputum Positive
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Did Not Meet Recommendations for Treatment With a Multidrug NTM Antimycobacterial Treatment
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
0
|
|
Overall Study
Withdrawal due to Covid-19
|
3
|
0
|
0
|
|
Overall Study
Sponsor decision to terminate study
|
3
|
2
|
4
|
Baseline Characteristics
Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial Infection
Baseline characteristics by cohort
| Measure |
Group 1
n=7 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=3 Participants
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=4 Participants
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
29.0 years
STANDARD_DEVIATION 11.78 • n=5 Participants
|
46.3 years
STANDARD_DEVIATION 20.03 • n=7 Participants
|
38.8 years
STANDARD_DEVIATION 12.28 • n=5 Participants
|
35.5 years
STANDARD_DEVIATION 14.62 • n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Causative species/ subspecies
Mycobacterium abscessus ss abscessus
|
6 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Causative species/ subspecies
Mycobacterium abscessus ss massiliense
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Causative species/ subspecies
Mycobacterium avium
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 48 weeksPopulation: Safety analysis set (participants who received at least one dose of trial treatment).
NTM sputum culture conversion to negative (defined as at least three consecutive negative mycobacterial cultures collected at least 4 weeks apart during the treatment period).
Outcome measures
| Measure |
Group 1
n=7 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=3 Participants
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=4 Participants
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Number of Participants With Sputum NTM Culture Conversion to Negative
|
3 Participants
|
1 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: Safety analysis set.
NTM sputum culture microbiological cure (defined as multiple consecutive negative but no positive cultures with the causative species after last culture conversion and until the end of treatment (Week 48)).
Outcome measures
| Measure |
Group 1
n=7 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=3 Participants
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=4 Participants
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Number of Participants With NTM Sputum Culture Microbiological Cure
|
1 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 48 WeeksPopulation: Only participants who had NTM sputum culture conversion during the treatment period were analyzed. The mean survival time and its standard error of the mean were underestimated because the largest observation was censored and the estimation was restricted to the largest event time.
Time to first NTM sputum culture conversion during the treatment period.
Outcome measures
| Measure |
Group 1
n=3 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=1 Participants
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=3 Participants
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Time to First NTM Sputum Culture Conversion
|
23.21 weeks
Standard Error 1.991
|
8.57 weeks
|
21.54 weeks
Standard Error 1.933
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: Safety analysis set. Only participants who had positive smear at baseline were analyzed.
Sputum smear conversion to negative (defined as at least three consecutive negative acid-fast bacilli stained sputum smears on microscopy, collected at least 4 weeks apart in subjects who were smear positive at baseline) during the treatment period.
Outcome measures
| Measure |
Group 1
n=2 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Number of Participants With Sputum Smear Conversion to Negative
|
2 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: Safety analysis set. Only participants who had positive smear at baseline were analyzed.
Consistent sputum smear conversion to negative (defined as multiple consecutive negative but no positive smears after last smear conversion and until the end of treatment (Week 48) in subjects who were smear positive at baseline).
Outcome measures
| Measure |
Group 1
n=2 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Number of Participants With Consistent Sputum Smear Conversion to Negative Treatment (Week 48) in Subjects Who Were Smear Positive at Baseline)
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: Safety analysis set. Only participants who had positive smear at baseline were analyzed. The mean survival time and its standard error were underestimated because the largest observation was censored and the estimation was restricted to the largest event time.
Time to first NTM sputum smear conversion during the treatment period.
Outcome measures
| Measure |
Group 1
n=2 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Time to First NTM Sputum Smear Conversion
|
14.55 weeks
Standard Error 2.084
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 weeks after end of treatmentPopulation: Safety analysis set. Only participants who had positive smear at baseline were analyzed.
Durable NTM sputum microbiological cure for the NTM isolate(s) treated without recurrence at Week 12 after end of treatment.
Outcome measures
| Measure |
Group 1
n=2 Participants
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Number of Participants With Durable NTM Sputum Microbiological Cure
|
0 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Group 1
Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths
Group 2
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Group 3
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group 1
n=7 participants at risk
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=3 participants at risk
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=4 participants at risk
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
14.3%
1/7 • Number of events 1 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Number of events 1 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Number of events 1 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
14.3%
1/7 • Number of events 1 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
Other adverse events
| Measure |
Group 1
n=7 participants at risk
Participants with chronic pulmonary MAC or MABSC infection who have not consistently achieved negative NTM sputum cultures while currently on a multidrug NTM guideline-based antimycobacterial regimen, which has been ongoing for at least 9 months prior to the Baseline visit.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 2
n=3 participants at risk
Participants with chronic pulmonary MAC or MABSC infection 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.
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
Group 3
n=4 participants at risk
Participants with chronic pulmonary MAC or MABSC infection not meeting recommendations for treatment with a multidrug NTM guideline-based antimycobacterial regimen based on failure to meet American Thoracic Society/Infectious Diseases Society of America (ATS/IDSA) criteria for NTM pulmonary disease (i.e. absence of radiologic findings and clinical symptoms beyond what is expected from underlying CF).
Molgramostim nebulizer solution: 300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
PARI eFlow nebulizer system: PARI eFlow nebulizer system
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
50.0%
2/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
66.7%
2/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Increased bronchial secretion
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal crusting
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Chest discomfort
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
66.7%
2/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
50.0%
2/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Fatigue
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Pyrexia
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Complication associated with device
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Feeling abnormal
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Pain
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Cystitis
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
General disorders
Localised infection
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Oral fungal infection
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Respiratory tract infection bacterial
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Staphylococcal infection
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Tooth infection
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Infections and infestations
Vaginal infection
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
White blood cell count decreased
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Blood glucose increased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Albumin urine present
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Blood bilirubin increased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Blood chloride decreased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Blood magnesium decreased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
C-reactive protein increased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Glucose urine present
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Specific gravity urine decreased
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Urine ketone body present
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Investigations
Weight increased
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Flatulence
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Nausea
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Parotid duct obstruction
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Nervous system disorders
Headache
|
28.6%
2/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Nervous system disorders
Dizziness
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Nervous system disorders
Paraesthesia
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Nervous system disorders
Sinus headache
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Psychiatric disorders
Depression
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Psychiatric disorders
Insomina
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Psychiatric disorders
Mania
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Renal and urinary disorders
Acute kidney injury
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Skin fissures
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Ear and labyrinth disorders
Ear pain
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Ear and labyrinth disorders
Vertigo
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Eye disorders
Photophobia
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
25.0%
1/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Metabolism and nutrition disorders
Dehydration
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
14.3%
1/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum discoloured
|
0.00%
0/7 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
33.3%
1/3 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
0.00%
0/4 • Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place