Trial Outcomes & Findings for Second Open Label Extension to Bridging Study CTBM100C2303 (NCT NCT01069705)
NCT ID: NCT01069705
Last Updated: 2021-06-02
Results Overview
An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug.
COMPLETED
PHASE3
49 participants
From time of first administration of study drug until study completion (up to 169 days)
2021-06-02
Participant Flow
The study was conducted at 14 centres in 8 countries from 12 February 2010 to 19 March 2012.
This study enrolled 49 Participants with Cystic Fibrosis who completed participation in the study CTBM100C2303E1 (NCT00982930).
Participant milestones
| Measure |
Tobramycin Inhalation Powder (TIPnew)
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Overall Study
STARTED
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49
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Overall Study
COMPLETED
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46
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Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Tobramycin Inhalation Powder (TIPnew)
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Overall Study
Adverse Event
|
1
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Overall Study
Withdrawal by Subject
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2
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Baseline Characteristics
Second Open Label Extension to Bridging Study CTBM100C2303
Baseline characteristics by cohort
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Age, Continuous
|
13.3 years
STANDARD_DEVIATION 4.31 • n=5 Participants
|
|
Sex: Female, Male
Female
|
32 Participants
n=5 Participants
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Sex: Female, Male
Male
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17 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: From time of first administration of study drug until study completion (up to 169 days)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study.
An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Number of Participants With Adverse Events (AEs)
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23 Participants
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PRIMARY outcome
Timeframe: From time of consent to 4 weeks after study completion (up to 199 days)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study.
A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Number of Participants With Serious Adverse Events (SAEs)
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2 Participants
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PRIMARY outcome
Timeframe: Pre-dose and post-dose of Day 1 and Day 29 of every Cycle (5, 6, 7)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Airway Reactivity \>= 20% relative decrease in FEV1% predicted from pre-dose to 30 minutes post-dose. Relative Change = 100 \* (30 minutes Post-dose - Pre-dose)/Pre-dose assessed by the number and percentage of participants with a decrease of ≥ 20% in FEV1 % predicted from pre-dose to 30 minutes post-dose.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 5 (Day 1)
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2.3 percentage of participants
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Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 5 (Day 29)
|
0.0 percentage of participants
|
|
Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 6 (Day 1)
|
2.5 percentage of participants
|
|
Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 6 (Day 29)
|
4.3 percentage of participants
|
|
Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 7 (Day 1)
|
6.7 percentage of participants
|
|
Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
Acute Change from Pre-dose to 30 mins Post-dose Cycle 7 (Day 29)
|
0.0 percentage of participants
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PRIMARY outcome
Timeframe: Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: All participants included in the safety population with at least one audiology testing. Number analyzed is the number of participants with data available at the given timepoint.
Auditory acuity of participants was measured using a standard dual-channel audiometer at frequencies from 250 to 8000 Hertz, and an audiogram (pure-tone air conduction) and tympanogram were performed by an audiologist. The categories reported includes \>= 10dB decrease in 3 consecutive frequencies in either ear, \>= 15dB decrease in 2 consecutive frequencies in either ear, and \>= 20dB decrease in at least one frequency in either ear
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=19 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 1; >= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
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0.0 percentage of participants
|
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 1; >= 15 dB Decrease in 2 Consecutive Frequencies in Either Ear
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0.0 percentage of participants
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 1; >= 20 dB Decrease in at least one Frequency in Either Ear
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0.0 percentage of participants
|
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 29; >= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
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0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 29; >= 15 dB Decrease in 2 Consecutive Frequencies in Either Ear
|
5.3 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 5: Day 29; >= 20 dB Decrease in at least one Frequency in Either Ear
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0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 6: Day 29; >= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
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0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 6: Day 29; >= 15 dB Decrease in 2 Consecutive Frequencies in Either Ear
|
5.3 percentage of participants
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 6: Day 29; >= 20 dB Decrease in at least one Frequency in Either Ear
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0.0 percentage of participants
|
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 7: Day 29; ˃= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
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10.5 percentage of participants
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 7: Day 29; >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
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5.3 percentage of participants
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Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 7: Day 29; >= 20 dB Decrease in at Least One Frequency in Either Ear
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5.3 percentage of participants
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|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Week 57/Day 57; ˃= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
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20.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Week 57/Day 57; >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
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0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Week 57/Day 57; >= 20 dB Decrease in at Least One Frequency in Either Ear
|
0.0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Forced expiratory volume in one second (FEV1) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEV1 % predicted was a normalized value of FEV1 calculated using the Knudsen equation, based upon participant's age, gender and height. Relative change in FEV1 % predicted from baseline to pre-dose day X = ((pre-dose day X FEV1 % predicted - baseline FEV1 % predicted) / baseline FEV1 % predicted) x 100.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Baseline
|
59.5 percent change in FEV1 % predicted
Standard Deviation 16.51
|
|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 1)
|
11.4 percent change in FEV1 % predicted
Standard Deviation 21.79
|
|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 29)
|
12.6 percent change in FEV1 % predicted
Standard Deviation 24.49
|
|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 1)
|
8.6 percent change in FEV1 % predicted
Standard Deviation 19.73
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|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 29)
|
11.9 percent change in FEV1 % predicted
Standard Deviation 24.23
|
|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 1)
|
9.0 percent change in FEV1 % predicted
Standard Deviation 22.44
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|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 29)
|
10.1 percent change in FEV1 % predicted
Standard Deviation 26.37
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|
Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Follow -Up (Week 57/Day 57)
|
8.1 percent change in FEV1 % predicted
Standard Deviation 25.79
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SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Percent Predicted Forced Vital Capacity (FVC%) is the maximal exhaled breath volume following a maximal inhaled breath. Overall change in percent predicted FVC = (observed value)/(predicted value) \* 100%. A higher value indicates a greater response.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
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Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Baseline
|
75.0 percent change in FVC % predicted
Standard Deviation 17.63
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 1)
|
5.1 percent change in FVC % predicted
Standard Deviation 15.60
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 29)
|
5.2 percent change in FVC % predicted
Standard Deviation 17.87
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 1)
|
2.6 percent change in FVC % predicted
Standard Deviation 16.36
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 29)
|
6.2 percent change in FVC % predicted
Standard Deviation 20.01
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 1)
|
2.3 percent change in FVC % predicted
Standard Deviation 19.20
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 29)
|
2.9 percent change in FVC % predicted
Standard Deviation 20.40
|
|
Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit
Relative Change from Baseline to Follow-up (Week 57/Day 57)
|
4.0 percent change in FVC % predicted
Standard Deviation 20.37
|
SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) is the forced expiratory flow from 25% to 75% of the Forced Vital Capacity (FVC). Relative change in FEF25-75% from baseline to pre-dose day X = (pre-dose day X FEF25-75 - baseline FEF25-75) / baseline FEF25-75) • 100.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
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|---|---|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Baseline
|
36.8 percent change in (FEF25-75)% predicted
Standard Deviation 20.30
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 1)
|
37.7 percent change in (FEF25-75)% predicted
Standard Deviation 57.39
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 5 (Day 29)
|
43.2 percent change in (FEF25-75)% predicted
Standard Deviation 67.51
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 1)
|
35.1 percent change in (FEF25-75)% predicted
Standard Deviation 66.39
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 6 (Day 29)
|
34.3 percent change in (FEF25-75)% predicted
Standard Deviation 63.53
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 1)
|
44.4 percent change in (FEF25-75)% predicted
Standard Deviation 80.91
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Cycle 7 (Day 29)
|
36.1 percent change in (FEF25-75)% predicted
Standard Deviation 61.75
|
|
Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit
Relative Change from Baseline to Follow-up (Week 57/Day 57)
|
35.6 percent change in (FEF25-75)% predicted
Standard Deviation 77.34
|
SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Pseudomonas Aeruginosa Density refers to overall density, defined as the sum of Biotypes (mucoid, dry and small colony variant). Absolute change was determined using the formula; Change = Post-baseline value- baseline value. Absolute Change in Pseudomonas Aeruginosa Sputum density is measured in log 10 Colony Forming Units per gram (Log 10 CFU/g).
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Baseline
|
7.4 Log 10 CFU/g
Standard Deviation 1.50
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 5 (Day 1)
|
-1.1 Log 10 CFU/g
Standard Deviation 2.82
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 5 (Day 29)
|
-3.7 Log 10 CFU/g
Standard Deviation 2.95
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 6 (Day 1)
|
-1.6 Log 10 CFU/g
Standard Deviation 2.95
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 6 (Day 29)
|
-3.6 Log 10 CFU/g
Standard Deviation 2.73
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 7 (Day 1)
|
-1.5 Log 10 CFU/g
Standard Deviation 3.40
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Cycle 7 (Day 29)
|
-2.6 Log 10 CFU/g
Standard Deviation 2.92
|
|
Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit
Change from Baseline to Follow-up (Week 57/Day 57)
|
-1.8 Log 10 CFU/g
Standard Deviation 3.62
|
SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
Minimum Inhibitory Concentration (MIC) is defined as the lowest concentration of an antimicrobial agent required to inhibit the visible growth of a microorganism after overnight incubation.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Baseline
|
2.2 microgram/millilitre
Standard Deviation 9.11
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 5 (Day 1)
|
31.4 microgram/millilitre
Standard Deviation 117.6
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 5 (Day 29)
|
21.6 microgram/millilitre
Standard Deviation 92.35
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 6 (Day 1)
|
39.3 microgram/millilitre
Standard Deviation 138.37
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 6 (Day 29)
|
25.5 microgram/millilitre
Standard Deviation 94.74
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 7 (Day 1)
|
3.7 microgram/millilitre
Standard Deviation 22.94
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline in Cycle 7 (Day 29)
|
31.7 microgram/millilitre
Standard Deviation 115.97
|
|
Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit
Change from Baseline to Follow-up (Week 57/Day 57)
|
1.7 microgram/millilitre
Standard Deviation 11.14
|
SECONDARY outcome
Timeframe: Baseline, Cycles 5, 6, 7 (Days 1, 29)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study.
The rate of anti-pseudomonal antibiotics use were determined from the collection of concomitant medication during the study Treatment period.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Number of Participants Who Used New Antipseudomonal Antibiotic During Treatment Period
|
7 Participants
|
SECONDARY outcome
Timeframe: From time of consent to 4 weeks after study completion (up to 199 days)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Percentage of Participants With Hospitalization Due to Respiratory Serious Adverse Events (SAEs)
|
4.1 percentage of participants
|
SECONDARY outcome
Timeframe: From time of consent to 4 weeks after study completion (up to 199 days)Population: Safety population included all participants who completed their participation in C2303E1, who gave their consent to enter the extension 2 study and who received at least one dose of study drug in the extension 2 study. Number analyzed is the number of participants with data available at the given timepoint.
The average number of days patients were hospitalized due to respiratory events during the study.
Outcome measures
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 Participants
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Number of Days of Hospitalization Due to Respiratory Serious Adverse Events (SAEs)
|
16.5 days
Standard Deviation 2.12
|
Adverse Events
Tobramycin Inhalation Powder (TIPnew)
Serious adverse events
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 participants at risk
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Pneumonia
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
Other adverse events
| Measure |
Tobramycin Inhalation Powder (TIPnew)
n=49 participants at risk
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
|
|---|---|
|
Ear and labyrinth disorders
Hypoacusis
|
4.1%
2/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Gastrointestinal disorders
Constipation
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Gastrointestinal disorders
Diarrhoea
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
General disorders
Asthenia
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
General disorders
Non-cardiac chest pain
|
4.1%
2/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
General disorders
Pyrexia
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Acute sinusitis
|
4.1%
2/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Alcaligenes infection
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Bronchitis
|
6.1%
3/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Burkholderia cepacia complex infection
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
4.1%
2/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Influenza
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Nasopharyngitis
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Respiratory tract infection
|
10.2%
5/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Infections and infestations
Respiratory tract infection viral
|
8.2%
4/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Investigations
Antibiotic resistant Staphylococcus test positive
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Investigations
Protein urine present
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.1%
3/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
4.1%
2/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
2.0%
1/49 • From the time of first administration of study drug until study completion (up to 169 days)
AEs were deemed treatment-emergent if the onset date was on or after the date of the first study drug of extension 2 and until study completion of the extension 2.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER