Trial Outcomes & Findings for Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections (NCT NCT01315678)
NCT ID: NCT01315678
Last Updated: 2020-06-16
Results Overview
Relative Change (%) from baseline to end of study (Day 168) in FEV1 (1 second)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
302 participants
Primary outcome timeframe
Baseline to168 days
Results posted on
2020-06-16
Participant Flow
302 participants were randomized. 8 were not treated.
Participant milestones
| Measure |
Arikayce™
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Overall Study
STARTED
|
148
|
146
|
|
Overall Study
COMPLETED
|
134
|
140
|
|
Overall Study
NOT COMPLETED
|
14
|
6
|
Reasons for withdrawal
| Measure |
Arikayce™
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
7
|
3
|
|
Overall Study
Adverse Event
|
3
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
similar reason to those listed above
|
3
|
2
|
Baseline Characteristics
Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Baseline characteristics by cohort
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
Total
n=294 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Age · 6-12 years
|
27 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
26 Participants
n=146 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
53 Participants
n=294 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Age, Customized
Age · >12-18 years
|
34 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
33 Participants
n=146 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
67 Participants
n=294 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Age, Customized
Age · >18 years
|
87 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
87 Participants
n=146 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
174 Participants
n=294 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Sex: Female, Male
Female
|
69 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
70 Participants
n=146 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
139 Participants
n=294 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Sex: Female, Male
Male
|
79 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
76 Participants
n=146 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
155 Participants
n=294 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Race/Ethnicity, Customized
Ethnicity · Caucasian
|
139 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
141 Participants
n=145 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
280 Participants
n=293 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Race/Ethnicity, Customized
Ethnicity · Hispanic
|
5 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
3 Participants
n=145 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
8 Participants
n=293 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Race/Ethnicity, Customized
Ethnicity · African
|
1 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
0 Participants
n=145 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
1 Participants
n=293 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
|
Race/Ethnicity, Customized
Ethnicity · Other
|
3 Participants
n=148 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
1 Participants
n=145 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
4 Participants
n=293 Participants • Modified intent-to-treat (mITT; received at least 1 dose of study drug)
|
PRIMARY outcome
Timeframe: Baseline to168 daysPopulation: The PP is the primary analysis population for the primary efficacy analysis
Relative Change (%) from baseline to end of study (Day 168) in FEV1 (1 second)
Outcome measures
| Measure |
Arikayce™
n=129 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=137 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Pulmonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
|
0.47 Percent (%) change
Standard Deviation 13.930
|
1.67 Percent (%) change
Standard Deviation 16.050
|
SECONDARY outcome
Timeframe: Baseline, Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168.Population: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Relative changes (%) from baseline to Study Days 14, 28, 57, 84, 113, 140, 168 in FEV1
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 57
|
-3.49 percentage (%) change
Standard Deviation 12.319
|
0.70 percentage (%) change
Standard Deviation 14.510
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 84
|
0.44 percentage (%) change
Standard Deviation 14.350
|
3.59 percentage (%) change
Standard Deviation 14.642
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 113
|
-0.90 percentage (%) change
Standard Deviation 12.028
|
0.42 percentage (%) change
Standard Deviation 14.434
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 140
|
0.43 percentage (%) change
Standard Deviation 15.299
|
1.37 percentage (%) change
Standard Deviation 16.563
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 14
|
2.59 percentage (%) change
Standard Deviation 13.332
|
6.64 percentage (%) change
Standard Deviation 15.669
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 28
|
0.79 percentage (%) change
Standard Deviation 14.745
|
3.32 percentage (%) change
Standard Deviation 14.715
|
|
Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1)
Day 168
|
-0.12 percentage (%) change
Standard Deviation 14.326
|
1.58 percentage (%) change
Standard Deviation 15.970
|
SECONDARY outcome
Timeframe: 168 daysPopulation: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Number of participants experiencing a pulmonary exacerbation measured by number with event and number censored
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Number of Participants Experiencing a Pulmonary Exacerbation
Number with Event
|
73 participants
|
63 participants
|
|
Number of Participants Experiencing a Pulmonary Exacerbation
Number Censored
|
75 participants
|
83 participants
|
SECONDARY outcome
Timeframe: 168 daysPopulation: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Number of participants to first antipseudomonal antibiotic treatment for pulmonary exacerbation measured by number with event and number censored
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Number of Participants to First Antipseudomonal Antibiotic Treatment for Pulmonary Exacerbation
Number with Event
|
55 Participants
|
48 Participants
|
|
Number of Participants to First Antipseudomonal Antibiotic Treatment for Pulmonary Exacerbation
Number Censored
|
93 Participants
|
98 Participants
|
SECONDARY outcome
Timeframe: 168 daysPopulation: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Number of participants to first all cause hospitalization measured by number with event and number censored
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Number of Participants to First All Cause Hospitalization
Number with Event
|
24 participants
|
29 participants
|
|
Number of Participants to First All Cause Hospitalization
Number Censored
|
124 participants
|
117 participants
|
SECONDARY outcome
Timeframe: Baseline, Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168Population: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Change in density (Log CFU) from baseline in Pseudomonas aeruginosa in sputum
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
Baseline
|
6.872 Log 10 CFU
Standard Deviation 1.8806
|
6.510 Log 10 CFU
Standard Deviation 2.3202
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
14 days
|
-1.124 Log 10 CFU
Standard Deviation 2.0542
|
-1.663 Log 10 CFU
Standard Deviation 2.4017
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
28 days
|
-1.208 Log 10 CFU
Standard Deviation 2.1594
|
-1.453 Log 10 CFU
Standard Deviation 2.4440
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
57 days
|
-0.210 Log 10 CFU
Standard Deviation 1.9874
|
-0.098 Log 10 CFU
Standard Deviation 1.7446
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
84 days
|
-0.945 Log 10 CFU
Standard Deviation 2.2223
|
-1.182 Log 10 CFU
Standard Deviation 2.6914
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
113 days
|
-0.613 Log 10 CFU
Standard Deviation 2.1928
|
-0.135 Log 10 CFU
Standard Deviation 2.3461
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
140 days
|
-1.440 Log 10 CFU
Standard Deviation 2.4357
|
-1.315 Log 10 CFU
Standard Deviation 2.2300
|
|
Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum
168 days
|
-0.725 Log 10 CFU
Standard Deviation 2.0073
|
-0.136 Log 10 CFU
Standard Deviation 2.1750
|
SECONDARY outcome
Timeframe: Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168Population: Modified intent-to-treat (mITT; received at least 1 dose of study drug)
Quality of Life was measured by the absolute change from baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory scale. Disease specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health. Scores for each Health Related Quality of Life (HRQoL) domain; after recoding, each item is summed to generate a domain score and standardized.
Outcome measures
| Measure |
Arikayce™
n=148 Participants
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 Participants
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 28
|
15.54 Percent (%) change
Standard Error 3.363
|
11.03 Percent (%) change
Standard Error 3.419
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 57
|
8.00 Percent (%) change
Standard Error 3.120
|
7.97 Percent (%) change
Standard Error 3.146
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 84
|
13.20 Percent (%) change
Standard Error 3.079
|
8.55 Percent (%) change
Standard Error 3.111
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 113
|
3.58 Percent (%) change
Standard Error 3.323
|
5.03 Percent (%) change
Standard Error 3.290
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 140
|
13.84 Percent (%) change
Standard Error 3.060
|
6.10 Percent (%) change
Standard Error 3.079
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 168
|
12.06 Percent (%) change
Standard Error 3.784
|
8.07 Percent (%) change
Standard Error 3.790
|
|
Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R
Day 14
|
13.65 Percent (%) change
Standard Error 2.995
|
8.81 Percent (%) change
Standard Error 3.019
|
Adverse Events
Arikayce™
Serious events: 26 serious events
Other events: 114 other events
Deaths: 0 deaths
TOBI®
Serious events: 29 serious events
Other events: 91 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Arikayce™
n=148 participants at risk
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 participants at risk
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.68%
1/148 • Number of events 1
|
0.68%
1/146 • Number of events 1
|
|
Gastrointestinal disorders
Dyspepsia
|
0.68%
1/148 • Number of events 1
|
0.68%
1/146 • Number of events 1
|
|
Gastrointestinal disorders
Haematochezia
|
0.00%
0/148
|
0.68%
1/146 • Number of events 1
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
16.2%
24/148 • Number of events 28
|
15.8%
23/146 • Number of events 28
|
|
Infections and infestations
Pneumonia
|
0.00%
0/148
|
1.4%
2/146 • Number of events 2
|
|
Infections and infestations
Bronchitis
|
0.00%
0/148
|
0.68%
1/146 • Number of events 1
|
|
Infections and infestations
Bronchopulmonary aspergillosis allergic
|
0.68%
1/148 • Number of events 1
|
0.00%
0/146
|
|
Infections and infestations
Chronic sinusitis
|
0.68%
1/148 • Number of events 1
|
0.00%
0/146
|
|
Investigations
Forced expiratory volume decreased
|
0.68%
1/148 • Number of events 1
|
0.00%
0/146
|
|
Investigations
Pulmonary function test decreased
|
0.00%
0/148
|
0.68%
1/146 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial secretion retention
|
0.68%
1/148 • Number of events 1
|
0.00%
0/146
|
|
Respiratory, thoracic and mediastinal disorders
Bronchiectasis
|
0.68%
1/148 • Number of events 1
|
0.00%
0/146
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/148
|
0.68%
1/146 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/148
|
0.68%
1/146 • Number of events 1
|
Other adverse events
| Measure |
Arikayce™
n=148 participants at risk
Arikayce™ is liposomal amikacin for inhalation
Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
* 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
* Administration time is approximately 13 minutes.
* Liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
|
TOBI®
n=146 participants at risk
TOBI® is tobramycin inhalation solution
Tobramycin inhalation solution using a PARI LC® Plus nebulizer: 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
* Nebulization time is approximately 20 minutes for each administration.
* Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
|
|---|---|---|
|
Gastrointestinal disorders
diarrhoea
|
6.8%
10/148 • Number of events 12
|
3.4%
5/146 • Number of events 5
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
55.4%
82/148 • Number of events 138
|
49.3%
72/146 • Number of events 117
|
|
Infections and infestations
Nasopharyngitis
|
16.2%
24/148 • Number of events 29
|
22.6%
33/146 • Number of events 43
|
|
Infections and infestations
Upper respiratory tract infection
|
10.1%
15/148 • Number of events 21
|
6.2%
9/146 • Number of events 14
|
|
Infections and infestations
Rhinitis
|
6.1%
9/148 • Number of events 10
|
6.2%
9/146 • Number of events 9
|
|
Nervous system disorders
Headache
|
8.1%
12/148 • Number of events 22
|
3.4%
5/146 • Number of events 8
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
16.2%
24/148 • Number of events 53
|
6.8%
10/146 • Number of events 21
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.2%
18/148 • Number of events 23
|
7.5%
11/146 • Number of events 24
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
12.2%
18/148 • Number of events 28
|
5.5%
8/146 • Number of events 9
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.4%
11/148 • Number of events 12
|
4.1%
6/146 • Number of events 7
|
|
General disorders
General disorders
|
13.5%
20/148 • Number of events 23
|
6.8%
10/146 • Number of events 13
|
|
Investigations
Investigations
|
8.1%
12/148 • Number of events 15
|
6.8%
10/146 • Number of events 11
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective
|
8.1%
12/148 • Number of events 14
|
6.8%
10/146 • Number of events 12
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous
|
5.4%
8/148 • Number of events 10
|
3.4%
5/146 • Number of events 6
|
Additional Information
Kevin Mange (Senior VP, Clinical Development and Medical Affairs)
Insmed Incorporated
Phone: 908-947-2651
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Per the signed Investigator Agreement in the study protocol and protocol amendments, the PI agreed "not to originate or use the name of Insmed Incorporated, or study drug code in any publicity, news release, or other public announcement, written or oral, whether to the public, press, or otherwise, relating to this protocol, to any amendment to the protocol, or to the performance of this protocol, without the prior written consent of Insmed Incorporated."
- Publication restrictions are in place
Restriction type: OTHER