Trial Outcomes & Findings for Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™) (NCT NCT00777296)
NCT ID: NCT00777296
Last Updated: 2020-07-30
Results Overview
Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
66 participants
Primary outcome timeframe
28 Days
Results posted on
2020-07-30
Participant Flow
Participant milestones
| Measure |
Cohort 1 - 280 mg ARIKACE™
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
Subjects in this arm of cohort 1 received matching placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this arm of cohort 2 received matching placebo
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
21
|
11
|
23
|
11
|
|
Overall Study
COMPLETED
|
20
|
10
|
21
|
10
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
2
|
1
|
Reasons for withdrawal
| Measure |
Cohort 1 - 280 mg ARIKACE™
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
Subjects in this arm of cohort 1 received matching placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this arm of cohort 2 received matching placebo
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
0
|
0
|
|
Overall Study
CRF form not completed
|
0
|
0
|
2
|
0
|
Baseline Characteristics
Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)
Baseline characteristics by cohort
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 Participants
Subjects in this arm of cohort 1 received matching placebo
|
Cohort 2 - 560 mg ARIKACE™
n=23 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 Participants
Subjects in this arm of cohort 2 received matching placebo
|
Total
n=66 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
16.0 years
STANDARD_DEVIATION 5.3 • n=5 Participants
|
16.9 years
STANDARD_DEVIATION 7.9 • n=7 Participants
|
16.6 years
STANDARD_DEVIATION 6.1 • n=5 Participants
|
17.2 years
STANDARD_DEVIATION 5.8 • n=4 Participants
|
16.6 years
STANDARD_DEVIATION 6.0 • n=21 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
39 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
27 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Caucasian (not of Hispanic origin)
|
21 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
66 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: 28 DaysPopulation: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug.
Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 Participants
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Clinically Significant Laboratory Abnormalities.
Neutrophils absolute
|
1 Participants
|
0 Participants
|
8 Participants
|
7 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Leucocytes
|
1 Participants
|
0 Participants
|
3 Participants
|
3 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Glucose
|
0 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Lymphocytes absolute
|
1 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Calcium
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Creatinine clearance
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Clinically Significant Laboratory Abnormalities.
Potassium
|
3 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 1, Day 14 and Day 28Population: The PK population consisted of subjects who received amikacin, had at least one serum PK assessment and were not replaced.
Measure PK parameters (AUC0-infinity) of Arikace™ in serum.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pharmacokinetics (PK) of Arikace™ in Serum.
Day 1
|
5.73 mg.hr/L
Standard Deviation 3.40
|
7.92 mg.hr/L
Standard Deviation 3.55
|
—
|
—
|
|
Pharmacokinetics (PK) of Arikace™ in Serum.
Day 14
|
7.61 mg.hr/L
Standard Deviation 4.04
|
12.5 mg.hr/L
Standard Deviation 10.9
|
—
|
—
|
|
Pharmacokinetics (PK) of Arikace™ in Serum.
Day 28
|
8.03 mg.hr/L
Standard Deviation 6.12
|
14.6 mg.hr/L
Standard Deviation 11.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1, Day 14 and Day 28Population: The PK population consisted of subjects who received amikacin, had at least one serum PK assessment and were not replaced.
Measure PK parameter (Cmax) of Arikace™ in serum.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Day 1
|
0.95 mg/L
Standard Deviation 0.58
|
1.08 mg/L
Standard Deviation 0.51
|
—
|
—
|
|
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Day 14
|
1.28 mg/L
Standard Deviation 1.02
|
1.84 mg/L
Standard Deviation 1.35
|
—
|
—
|
|
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Day 28
|
1.42 mg/L
Standard Deviation 1.45
|
2.27 mg/L
Standard Deviation 1.58
|
—
|
—
|
SECONDARY outcome
Timeframe: 28 daysPopulation: The PK population consisted of subjects who received amikacin, had at least one serum PK assessment and were not replaced.
Measure PK parameter (AUC0-24) of Arikace™ in sputum.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pharmacokinetics (PK) of Arikace™ in Sputum (AUC).
|
13120 mcg*hr/g
Standard Deviation 21386
|
22445 mcg*hr/g
Standard Deviation 18652
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1, Day 14 and Day 28Population: The PK population consisted of subjects who received amikacin, had at least one serum PK assessment and were not replaced.
Measure PK parameter (Ae0-24 (mg) of Arikace™.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pharmacokinetics (PK) of Arikace™ in Urine.
Day 1
|
17.7 mg
Standard Deviation 12.3
|
27.0 mg
Standard Deviation 25.2
|
—
|
—
|
|
Pharmacokinetics (PK) of Arikace™ in Urine.
Day 14
|
27.3 mg
Standard Deviation 16.5
|
39.8 mg
Standard Deviation 42.7
|
—
|
—
|
|
Pharmacokinetics (PK) of Arikace™ in Urine.
Day 28
|
25.2 mg
Standard Deviation 19.5
|
43.7 mg
Standard Deviation 48.9
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1, Day 14 and Day 28Population: The PK population consisted of subjects who received amikacin, had at least one serum PK assessment and were not replaced.
Measure PK parameter (sputum amicakin concentration) of Arikace™ in sputum.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Sputum Amikacin Levels of Arikace™.
Day 1
|
1197 mcg/g
Standard Deviation 1.56
|
2395 mcg/g
Standard Deviation 0.866
|
—
|
—
|
|
Sputum Amikacin Levels of Arikace™.
Day 14
|
1174 mcg/g
Standard Deviation 1.01
|
3496 mcg/g
Standard Deviation 0.973
|
—
|
—
|
|
Sputum Amikacin Levels of Arikace™.
Day 28
|
1911 mcg/g
Standard Deviation 1.28
|
2635 mcg/g
Standard Deviation 1.23
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, Day 28, and Day 56Population: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug. Placebo is a pooled value from cohort 280 mg and cohort 560 mg.
Relative Change (%) from Baseline to End of treatment (Day 28) and Day 56 in Pulmonary Function.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=22 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pulmonary Function: FEV1 %-Predicted.
Baseline
|
66.4 Relative Percent (%) change in FEV1
Standard Deviation 20.0
|
62.9 Relative Percent (%) change in FEV1
Standard Deviation 18.2
|
68.0 Relative Percent (%) change in FEV1
Standard Deviation 22.4
|
—
|
|
Pulmonary Function: FEV1 %-Predicted.
Day 28
|
9.6 Relative Percent (%) change in FEV1
Standard Deviation 13.7
|
11.0 Relative Percent (%) change in FEV1
Standard Deviation 16.4
|
0.5 Relative Percent (%) change in FEV1
Standard Deviation 10.5
|
—
|
|
Pulmonary Function: FEV1 %-Predicted.
Day 56
|
1.8 Relative Percent (%) change in FEV1
Standard Deviation 8.8
|
13.8 Relative Percent (%) change in FEV1
Standard Deviation 26.2
|
-3.8 Relative Percent (%) change in FEV1
Standard Deviation 13.5
|
—
|
SECONDARY outcome
Timeframe: Baseline, Day 28, and Day 56Population: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug. Placebo is a pooled value from cohort 280 mg and cohort 560 mg.
Mean Percent Change (%) from Baseline to End of treatment (Day 28) and Day 56 in Pulmonary Function.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=22 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Pulmonary Function: FEV1.
Baseline
|
2.022 Mean Percent (%) Change in FEV1
Standard Deviation 0.788
|
1.937 Mean Percent (%) Change in FEV1
Standard Deviation 0.936
|
1.968 Mean Percent (%) Change in FEV1
Standard Deviation 0.654
|
—
|
|
Pulmonary Function: FEV1.
Day 28
|
10.1 Mean Percent (%) Change in FEV1
Standard Deviation 12.8
|
13.2 Mean Percent (%) Change in FEV1
Standard Deviation 16.2
|
2.2 Mean Percent (%) Change in FEV1
Standard Deviation 11.9
|
—
|
|
Pulmonary Function: FEV1.
Day 56
|
2.0 Mean Percent (%) Change in FEV1
Standard Deviation 8.6
|
13.2 Mean Percent (%) Change in FEV1
Standard Deviation 24.3
|
-4.4 Mean Percent (%) Change in FEV1
Standard Deviation 13.0
|
—
|
SECONDARY outcome
Timeframe: Day 7, Day 14, Day 21, Day 28 and Day 35Population: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug.
End-of-treatment (Day 28) from baseline in density of P. aeruginosa (log10 CFU/g) in sputum.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=21 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=22 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Day 7
|
0.080 log10CFU per gram
Standard Deviation 1.882
|
-1.101 log10CFU per gram
Standard Deviation 2.170
|
0.052 log10CFU per gram
Standard Deviation 1.303
|
—
|
|
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Day 14
|
-1.366 log10CFU per gram
Standard Deviation 2.013
|
-1.570 log10CFU per gram
Standard Deviation 2.161
|
-0.574 log10CFU per gram
Standard Deviation 1.006
|
—
|
|
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Day 21
|
-1.044 log10CFU per gram
Standard Deviation 2.155
|
-2.283 log10CFU per gram
Standard Deviation 2.775
|
-0.440 log10CFU per gram
Standard Deviation 1.280
|
—
|
|
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Day 28
|
-0.622 log10CFU per gram
Standard Deviation 1.881
|
-1.515 log10CFU per gram
Standard Deviation 1.699
|
-0.677 log10CFU per gram
Standard Deviation 1.043
|
—
|
|
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Day 35
|
-0.380 log10CFU per gram
Standard Deviation 1.425
|
-1.313 log10CFU per gram
Standard Deviation 2.852
|
-0.445 log10CFU per gram
Standard Deviation 1.201
|
—
|
SECONDARY outcome
Timeframe: Through study duration, approximately 56 daysPopulation: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug.
Duration of systemic antipseudomonal rescue therapy during the study in both the ARIKACE™ and placebo groups.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 Participants
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Duration of Systemic Antipseudomonal Rescue Therapy.
|
14.00 days
Standard Deviation 0.00
|
27.00 days
Standard Deviation NA
Standard deviation does not apply because only 1 participant required rescue therapy
|
19.00 days
Standard Deviation NA
Standard deviation does not apply because only 1 participant required rescue therapy
|
21.00 days
Standard Deviation 11.31
|
SECONDARY outcome
Timeframe: Through study duration, approximately 56 daysPopulation: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug.
Number of Subjects requiring systemic antipseudomonal rescue therapy during the study in both the ARIKACE™ and placebo groups.
Outcome measures
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=42 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=22 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
Number of Subjects Requiring Antipseudomonal Rescue Therapy.
|
4 Participants
|
3 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline/Day 1, Day 15, Day 28 and Day 42Population: The analysis population is the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one 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 |
Cohort 1 - 280 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 Participants
Subjects in this cohort who received placebo
|
Cohort 2 - 560 mg ARIKACE™
n=21 Participants
Subjects in this cohort received 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 Participants
Subjects in this cohort received matching placebo
|
|---|---|---|---|---|
|
CFQ-R Respiratory Scale (Absolute Change From Baseline).
Day 1/baseline
|
72.619 score on a scale
Standard Deviation 11.630
|
71.212 score on a scale
Standard Deviation 14.921
|
67.989 score on a scale
Standard Deviation 12.748
|
61.364 score on a scale
Standard Deviation 21.425
|
|
CFQ-R Respiratory Scale (Absolute Change From Baseline).
Day 15
|
2.632 score on a scale
Standard Deviation 10.078
|
-0.505 score on a scale
Standard Deviation 12.349
|
3.704 score on a scale
Standard Deviation 16.133
|
-2.778 score on a scale
Standard Deviation 16.054
|
|
CFQ-R Respiratory Scale (Absolute Change From Baseline).
Day 28
|
4.306 score on a scale
Standard Deviation 12.760
|
-3.283 score on a scale
Standard Deviation 14.154
|
5.688 score on a scale
Standard Deviation 11.669
|
1.667 score on a scale
Standard Deviation 13.302
|
|
CFQ-R Respiratory Scale (Absolute Change From Baseline).
Day 42
|
1.080 score on a scale
Standard Deviation 12.169
|
-4.012 score on a scale
Standard Deviation 19.598
|
3.042 score on a scale
Standard Deviation 18.213
|
0.556 score on a scale
Standard Deviation 12.200
|
Adverse Events
Cohort 1 - 280 mg ARIKACE™
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Cohort 1 - Placebo
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Cohort 2 - 560 mg ARIKACE™
Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths
Cohort 2 - Placebo
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 participants at risk
Subjects in this cohort will receive 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 participants at risk
Subjects in this arm of cohort 1 will receive matching placebo
|
Cohort 2 - 560 mg ARIKACE™
n=21 participants at risk
Subjects in this cohort will receive 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 participants at risk
Subjects in this arm of cohort 2 will receive matching placebo
|
|---|---|---|---|---|
|
Infections and infestations
ENTEROVIRAL INFECTION
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EXACERBATION
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
Other adverse events
| Measure |
Cohort 1 - 280 mg ARIKACE™
n=21 participants at risk
Subjects in this cohort will receive 280 mg of ARIKACE™
|
Cohort 1 - Placebo
n=11 participants at risk
Subjects in this arm of cohort 1 will receive matching placebo
|
Cohort 2 - 560 mg ARIKACE™
n=21 participants at risk
Subjects in this cohort will receive 560 mg of ARIKACE™
|
Cohort 2 - Placebo
n=11 participants at risk
Subjects in this arm of cohort 2 will receive matching placebo
|
|---|---|---|---|---|
|
Gastrointestinal disorders
APHTHOUS STOMATITIS
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
General disorders
PYREXIA
|
4.8%
1/21 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
18.2%
2/11 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Infections and infestations
NASOPHARYNGITIS
|
4.8%
1/21 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
4.8%
1/21 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Infections and infestations
PHARYNGITIS
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Infections and infestations
SINUSITIS
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Investigations
NEUTROPHIL COUNT DECREASED
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Nervous system disorders
HEADACHE
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Nervous system disorders
SYNCOPE
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
ASTHMA
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
HAEMOPTYSIS
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
LUNG DISORDER
|
4.8%
1/21 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
PRODUCTIVE COUGH
|
14.3%
3/21 • Number of events 4 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Respiratory, thoracic and mediastinal disorders
RHINITIS ALLERGIC
|
9.5%
2/21 • Number of events 2 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
|
Skin and subcutaneous tissue disorders
URTICARIA
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
9.1%
1/11 • Number of events 1 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/21 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
0.00%
0/11 • AEs were assessed from the first dose (Visit 2) until the completion of the study follow-up (14 days after 28 days of dosing in cohort 2). The total duration is approximately 84 days.
|
Additional Information
Dr. Kevin Mange, Sr. Vice President, Clinical Development
Insmed Incorporated
Phone: 908-947-2651
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER