Trial Outcomes & Findings for A Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI® (NCT NCT00885365)

NCT ID: NCT00885365

Last Updated: 2018-06-27

Results Overview

Pulmonary function measurements were performed by using a self-calibrated computer-operated pneumotochographic spirometer at all clinic visits. Because this study included both adults and children and lung volume is an age-dependent variable, reference normal FEV1 values for children were different than the reference normal values used with adult participants. Three measurements were collected and the greatest FEV1 value was recorded.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

324 participants

Primary outcome timeframe

Day 0 (baseline), Week 4

Results posted on

2018-06-27

Participant Flow

406 participants screened, 324 participants randomized

Participant milestones

Participant milestones
Measure
Bramitob
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
tobramycin / TOBI administered 300mg twice a day for 4 weeks
4 Weeks ON Treatment
STARTED
159
165
4 Weeks ON Treatment
Intent-to-Treat Population
158
163
4 Weeks ON Treatment
Safety Population
156
168
4 Weeks ON Treatment
COMPLETED
155
159
4 Weeks ON Treatment
NOT COMPLETED
4
6
4 Weeks OFF Treatment
STARTED
155
159
4 Weeks OFF Treatment
COMPLETED
155
159
4 Weeks OFF Treatment
NOT COMPLETED
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Bramitob
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
tobramycin / TOBI administered 300mg twice a day for 4 weeks
4 Weeks ON Treatment
Adverse Event
2
4
4 Weeks ON Treatment
Protocol Violation
1
2
4 Weeks ON Treatment
Withdrawal by Subject
1
0

Baseline Characteristics

A Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI®

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=168 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Total
n=324 Participants
Total of all reporting groups
Age, Continuous
15.95 years
STANDARD_DEVIATION 6.74 • n=93 Participants
15.74 years
STANDARD_DEVIATION 7.24 • n=4 Participants
15.84 years
STANDARD_DEVIATION 6.99 • n=27 Participants
Age, Customized
6-12 years
49 participants
n=93 Participants
54 participants
n=4 Participants
103 participants
n=27 Participants
Age, Customized
13-17 years
50 participants
n=93 Participants
62 participants
n=4 Participants
112 participants
n=27 Participants
Age, Customized
> 17 years
57 participants
n=93 Participants
52 participants
n=4 Participants
109 participants
n=27 Participants
Sex: Female, Male
Female
84 Participants
n=93 Participants
83 Participants
n=4 Participants
167 Participants
n=27 Participants
Sex: Female, Male
Male
72 Participants
n=93 Participants
85 Participants
n=4 Participants
157 Participants
n=27 Participants
Race/Ethnicity, Customized
White
156 participants
n=93 Participants
168 participants
n=4 Participants
324 participants
n=27 Participants
Height
153.05 centimeters
STANDARD_DEVIATION 17.31 • n=93 Participants
153.41 centimeters
STANDARD_DEVIATION 19.88 • n=4 Participants
153.24 centimeters
STANDARD_DEVIATION 18.66 • n=27 Participants
Weight
42.64 kilograms
STANDARD_DEVIATION 14.92 • n=93 Participants
43.81 kilograms
STANDARD_DEVIATION 16.39 • n=4 Participants
43.25 kilograms
STANDARD_DEVIATION 15.69 • n=27 Participants
Body Mass Index
17.55 kilograms/meters^2
STANDARD_DEVIATION 3.12 • n=93 Participants
17.79 kilograms/meters^2
STANDARD_DEVIATION 3.32 • n=4 Participants
17.68 kilograms/meters^2
STANDARD_DEVIATION 3.22 • n=27 Participants
Time from diagnosis of chronic colonization of P. aeruginosa
2.27 years
STANDARD_DEVIATION 4.22 • n=93 Participants
2.36 years
STANDARD_DEVIATION 4.23 • n=4 Participants
2.31 years
STANDARD_DEVIATION 4.22 • n=27 Participants
Time from first Cystic Fibrosis diagnosis
12.16 years
STANDARD_DEVIATION 6.13 • n=93 Participants
11.74 years
STANDARD_DEVIATION 6.55 • n=4 Participants
11.94 years
STANDARD_DEVIATION 6.34 • n=27 Participants
Tobramycin Minimal Inhibitory Concentration (MIC) value
<16 mcg/mL
143 participants
n=93 Participants
159 participants
n=4 Participants
302 participants
n=27 Participants
Tobramycin Minimal Inhibitory Concentration (MIC) value
>=16 mcg/mL
13 participants
n=93 Participants
8 participants
n=4 Participants
21 participants
n=27 Participants
Tobramycin Minimal Inhibitory Concentration (MIC) value
Missing
0 participants
n=93 Participants
1 participants
n=4 Participants
1 participants
n=27 Participants
Forced Expiratory Volume in 1 second (FEV1)
<50 % predicted
37 participants
n=93 Participants
38 participants
n=4 Participants
75 participants
n=27 Participants
Forced Expiratory Volume in 1 second (FEV1)
>=50 % predicted
119 participants
n=93 Participants
130 participants
n=4 Participants
249 participants
n=27 Participants
Use of rhDNase
Yes
110 participants
n=93 Participants
119 participants
n=4 Participants
229 participants
n=27 Participants
Use of rhDNase
No
46 participants
n=93 Participants
49 participants
n=4 Participants
95 participants
n=27 Participants

PRIMARY outcome

Timeframe: Day 0 (baseline), Week 4

Population: Intent-to-Treat (ITT) Population, Last Observation Carried Forward (LOCF)

Pulmonary function measurements were performed by using a self-calibrated computer-operated pneumotochographic spirometer at all clinic visits. Because this study included both adults and children and lung volume is an age-dependent variable, reference normal FEV1 values for children were different than the reference normal values used with adult participants. Three measurements were collected and the greatest FEV1 value was recorded.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of the Treatment Period of Forced Expiratory Volume in 1 Second (FEV1), Expressed as Percentage of Predicted Normal
6.99 percentage predicted FEV1
Standard Deviation 9.52
7.51 percentage predicted FEV1
Standard Deviation 9.63

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FEV1 test.

Pulmonary function measurements were performed by using a self-calibrated computer-operated pneumotochographic spirometer at all clinic visits. Because this study included both adults and children and lung volume is an age-dependent variable, reference normal FEV1 values for children were different than the reference normal values used with adult participants. Three measurements were collected and the greatest FEV1 value was recorded. Observed values are summarized (without last observation carry forward).

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Volume in 1 Second (FEV1), Expressed as Percentage of Predicted Normal
Week 2 [n=158, 163]
6.70 percentage predicted FEV1
Standard Deviation 9.99
6.93 percentage predicted FEV1
Standard Deviation 9.40
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Volume in 1 Second (FEV1), Expressed as Percentage of Predicted Normal
Week 4 [N=155, 161]
7.10 percentage predicted FEV1
Standard Deviation 9.57
7.63 percentage predicted FEV1
Standard Deviation 9.61
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Volume in 1 Second (FEV1), Expressed as Percentage of Predicted Normal
Week 8 [n=155, 159]
5.47 percentage predicted FEV1
Standard Deviation 11.88
5.37 percentage predicted FEV1
Standard Deviation 11.11

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FEV1 test.

Pulmonary function measurements were performed by using a self-calibrated computer-operated pneumotochographic spirometer at all clinic visits. Three measurements were collected and the greatest FEV1 value was recorded. Observed values are summarized (without last observation carry forward).

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Expiratory Volume in 1 Second (FEV1)
Week 2 [n=158, 163]
0.18 liters
Standard Deviation 0.27
0.19 liters
Standard Deviation 0.27
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Expiratory Volume in 1 Second (FEV1)
Week 4 [n=155, 161]
0.20 liters
Standard Deviation 0.29
0.22 liters
Standard Deviation 0.28
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Expiratory Volume in 1 Second (FEV1)
Week 8 [n=155,159]
0.16 liters
Standard Deviation 0.33
0.16 liters
Standard Deviation 0.32

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FVC test.

FVC is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. It is measured via spirometry. Because this study included both adults and children and lung volume is an age-dependent variable, reference normal FVC values for children were different than the reference normal values used with adult participants.

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline at End of Weeks 2, 4, and 8 of Forced Vital Capacity (FVC) Expressed as Percentage of Predicted Normal
Week 8 [n=153,159]
2.70 percentage predicted FVC
Standard Deviation 12.30
5.04 percentage predicted FVC
Standard Deviation 12.21
Change From Baseline at End of Weeks 2, 4, and 8 of Forced Vital Capacity (FVC) Expressed as Percentage of Predicted Normal
Week 2 [n=156, 163]
4.36 percentage predicted FVC
Standard Deviation 10.81
5.36 percentage predicted FVC
Standard Deviation 10.03
Change From Baseline at End of Weeks 2, 4, and 8 of Forced Vital Capacity (FVC) Expressed as Percentage of Predicted Normal
Week 4 [n=153, 161]
4.83 percentage predicted FVC
Standard Deviation 10.05
5.58 percentage predicted FVC
Standard Deviation 10.86

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FVC test.

FVC is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. It is measured via spirometry.

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Vital Capacity (FVC)
Week 2 [n=156, 163]
0.14 liters
Standard Deviation 0.34
0.16 liters
Standard Deviation 0.32
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Vital Capacity (FVC)
Week 4 [n=153, 161]
0.16 liters
Standard Deviation 0.34
0.18 liters
Standard Deviation 0.36
Change From Baseline to End of Weeks 2, 4, and 8 of Absolute Forced Vital Capacity (FVC)
Week 8 [n=153,159]
0.10 liters
Standard Deviation 0.38
0.16 liters
Standard Deviation 0.39

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FEF 25-75% test.

Difference in the forced expiratory flow rate in mid-exhalation as a percent of predicted to standard values measured from baseline to weeks 2 (treated), 4 (treated), and 8 (untreated). Because this study included both adults and children and lung volume is an age-dependent variable, reference normal FEF 25-75% values for children were different than the reference normal values used with adult participants.

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=162 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%), Expressed as Percentage of Predicted Normal
Week 2 [n=156, 162]
10.30 percentage predicted FEF 25-75%
Standard Deviation 17.07
8.57 percentage predicted FEF 25-75%
Standard Deviation 19.00
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%), Expressed as Percentage of Predicted Normal
Week 4 [n=154, 159]
10.44 percentage predicted FEF 25-75%
Standard Deviation 15.17
9.32 percentage predicted FEF 25-75%
Standard Deviation 17.99
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%), Expressed as Percentage of Predicted Normal
Week 8 [n=154,158]
9.05 percentage predicted FEF 25-75%
Standard Deviation 19.59
6.41 percentage predicted FEF 25-75%
Standard Deviation 20.46

SECONDARY outcome

Timeframe: Day 0 (baseline), Week 2, Week 4, Week 8

Population: Intent-to-Treat (ITT) Population, observed values. Differences in number of participants analyzed to the ITT population represent participants who missed visits, or failed to take the FEF 25-75% test.

Difference in the forced expiratory flow rate in mid-exhalation measured from baseline to weeks 2 (treated), 4 (treated), and 8 (untreated).

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=162 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%)
Week 2 [n=156, 162]
0.32 liters/second
Standard Deviation 0.50
0.28 liters/second
Standard Deviation 0.56
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%)
Week 4 [n=154, 159]
0.34 liters/second
Standard Deviation 0.48
0.29 liters/second
Standard Deviation 0.57
Change From Baseline to End of Weeks 2, 4, and 8 of Forced Expiratory Flow at 25-75% of Vital Capacity (FEF 25-75%)
Week 8 [n=154,158]
0.28 liters/second
Standard Deviation 0.57
0.21 liters/second
Standard Deviation 0.67

SECONDARY outcome

Timeframe: Day -10 to -1 (baseline), Week 4, Week 8

Population: Intent-to-Treat (ITT) Population; At Week 4, six Bramitob patients and seven TOBI patients were missing sputum samples. At Week 8, 11 Bramitob patients and 16 TOBI patients were missing sputum samples.

If a participant had more than one Pseudomonas aeruginosa (PA) morphotype at a given visit, and therefore more than one bacterial load value, then the bacterial load value corresponding to the highest tobramycin minimal inhibitory concentration (MIC) value regardless of the PA morphotype was used. If the tobramycin MIC value was the same for different PA morphotypes, then the bacterial load value corresponding to morphotype 1 (mucoid colony) was used. If morphotype 1 was not available, bacterial load value corresponding to morphotype 2 (dry colony) was used.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 4 and 8 in Pseudomonas Aeruginosa Log10 Bacterial Load in Sputum
Week 4 [n=152, 156]
-2.14 colony forming units/gram
Standard Deviation 2.41
-2.07 colony forming units/gram
Standard Deviation 2.20
Change From Baseline to End of Weeks 4 and 8 in Pseudomonas Aeruginosa Log10 Bacterial Load in Sputum
Week 8 [n=147, 147]
-0.72 colony forming units/gram
Standard Deviation 2.17
-0.87 colony forming units/gram
Standard Deviation 2.23

SECONDARY outcome

Timeframe: Week 4, Week 8

Population: Intent-to-Treat (ITT) Population

MIC50 is the concentration of tobramycin required to inhibit 50% of Pseudomonas aeruginosa. MIC values were calculated for three different Pseudomonas aeruginosa (PA) strains: * Morphotype 1: mucoid * Morphotype 2: dry * Morphotype 3: variant Overall MIC50 values are reported. If a participant has more than one PA morphotype at a given visit, then the highest tobramycin MIC value was used, regardless of PA morphotype. If a participant has more than one available result for each morphotype then the highest tobramycin MIC value was used. If the tobramycin MIC values are equal then the MIC value for the isolate with the highest bacterial load value was used.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Minimal Inhibitory Concentration Inhibiting Growth of 50% (MIC50) of Pseudomonas Aeruginosa
Week 4 [n=126, 134]
1 micrograms/milliliters
0.5 micrograms/milliliters
Minimal Inhibitory Concentration Inhibiting Growth of 50% (MIC50) of Pseudomonas Aeruginosa
Week 8 [n=129, 128]
1 micrograms/milliliters
1 micrograms/milliliters

SECONDARY outcome

Timeframe: Week 4, Week 8

Population: Intent-to-Treat (ITT) Population

MIC90 is the concentration of tobramycin required to inhibit 90% of Pseudomonas aeruginosa. MIC values were calculated for three different Pseudomonas aeruginosa (PA) strains: * Morphotype 1: mucoid * Morphotype 2: dry * Morphotype 3: variant Overall MIC90 values are reported. If a participant has more than one PA morphotype at a given visit, then the highest tobramycin MIC value was used, regardless of PA morphotype. If a participant has more than one available result for each morphotype then the highest tobramycin MIC value was used. If the tobramycin MIC values are equal then the MIC value for the isolate with the highest bacterial load value was used.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Minimal Inhibitory Concentration Inhibiting Growth of 90% (MIC90) of Pseudomonas Aeruginosa
Week 4 [n=126, 134]
32 micrograms/milliliters
32 micrograms/milliliters
Minimal Inhibitory Concentration Inhibiting Growth of 90% (MIC90) of Pseudomonas Aeruginosa
Week 8 [n=129, 128]
32 micrograms/milliliters
32 micrograms/milliliters

SECONDARY outcome

Timeframe: Day -10 to -1 (screening), Weeks 4 and 8

Population: Intent-to-Treat Population.

Microbiological outcomes are derived considering all P. aeruginosa (PA) morphotypes together. Week 4 and Week 8 microbiological outcomes: * Eradication = elimination of PA * Persistence = persistence of PA detected at previous visit * Superinfection = appearance of a pathogen (other than PA) not detected at previous visit * Re-infection (week 8 only) = re-appearance of PA detected at Screening and eradicated at Week 4 Superinfection supersedes eradication. Persistence for P. aeruginosa supersedes superinfection. Re-infection for P. aeruginosa supersedes superinfection.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Microbiological Outcome Summary by Visit
Screening: Presence of P aeruginosa
100 percentage of participants
100 percentage of participants
Microbiological Outcome Summary by Visit
Week 4: Eradication
9.2 percentage of participants
7.1 percentage of participants
Microbiological Outcome Summary by Visit
Week 4: Persistence
82.9 percentage of participants
85.3 percentage of participants
Microbiological Outcome Summary by Visit
Week 4: Superinfection
7.9 percentage of participants
7.7 percentage of participants
Microbiological Outcome Summary by Visit
Week 8: Eradication
2.7 percentage of participants
3.4 percentage of participants
Microbiological Outcome Summary by Visit
Week 8: Persistence
78.9 percentage of participants
83.0 percentage of participants
Microbiological Outcome Summary by Visit
Week 8: Superinfection
9.5 percentage of participants
9.5 percentage of participants
Microbiological Outcome Summary by Visit
Week 8: Re-infection
8.8 percentage of participants
4.1 percentage of participants

SECONDARY outcome

Timeframe: Day 0 (baseline), Weeks 2, 4 and 8

Population: Intent-to-Treat (ITT) Population, observed values. Two participants from both treatment arms were missing data at Week 4. Three participants from Bramitob and 4 participants from TOBI were missing data at Week 8.

Body weight was measured at all study visits as part of the physical examination.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4 and 8 in Body Weight
Week 2 [n=158, 163]
0.24 kilograms
Standard Deviation 0.68
0.22 kilograms
Standard Deviation 0.72
Change From Baseline to End of Weeks 2, 4 and 8 in Body Weight
Week 4 [n=156, 161]
0.39 kilograms
Standard Deviation 0.90
0.42 kilograms
Standard Deviation 0.97
Change From Baseline to End of Weeks 2, 4 and 8 in Body Weight
Week 8 [n=155,159]
0.60 kilograms
Standard Deviation 1.14
0.53 kilograms
Standard Deviation 1.27

SECONDARY outcome

Timeframe: Day 0 (baseline), Weeks 2, 4 and 8

Population: Intent-to-Treat (ITT) Population, observed values. Two participants from both treatment arms were missing data at Week 4. Three participants from Bramitob and 4 participants from TOBI were missing data at Week 8.

Outcome measures

Outcome measures
Measure
Bramitob
n=158 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=163 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Change From Baseline to End of Weeks 2, 4 and 8 in Body Mass Index (BMI)
Week 2 [n=158, 163]
0.10 kilograms/meters^2
Standard Deviation 0.28
0.10 kilograms/meters^2
Standard Deviation 0.29
Change From Baseline to End of Weeks 2, 4 and 8 in Body Mass Index (BMI)
Week 4 [n=156, 161]
0.12 kilograms/meters^2
Standard Deviation 0.37
0.13 kilograms/meters^2
Standard Deviation 0.38
Change From Baseline to End of Weeks 2, 4 and 8 in Body Mass Index (BMI)
Week 8 [n=155,159]
0.16 kilograms/meters^2
Standard Deviation 0.48
0.14 kilograms/meters^2
Standard Deviation 0.49

SECONDARY outcome

Timeframe: Day 0 to Week 8

Population: Safety population: all randomised patients who took at least one dose of study medication

Treatment-Emergent Adverse Events defined as adverse events occurring after the first intake of study treatment (or the same day). The investigator assessed relation to study treatment as a binary question: Reasonable possibility of relatedness or no reasonable possibility of relatedness. The expression "reasonable possibility of relatedness" is meant to convey in general that there are facts (evidence) or arguments meant to suggest a causal relationship. A serious AE results in death, is life-threatening, requires hospitalization or prolongation of existing inpatient hospitalization, results in persistent or significant disability or incapacity, is a congenital anomaly/birth defect or an important medical event. The investigator rates the severity of the AE based on a three point scale: mild, moderate or severe. A severe event prevents any usual routine activity of the participant and causes severe discomfort.

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=168 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Count of Participants With Treatment-Emergent Adverse Events (TEAEs)
Severe TEAE
1.9 percentage of participants
1.2 percentage of participants
Count of Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAE
31.4 percentage of participants
28.0 percentage of participants
Count of Participants With Treatment-Emergent Adverse Events (TEAEs)
Related TEAE
6.4 percentage of participants
6.0 percentage of participants
Count of Participants With Treatment-Emergent Adverse Events (TEAEs)
Serious TEAE
3.8 percentage of participants
1.2 percentage of participants
Count of Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAE leading to withdrawal
0.6 percentage of participants
3.0 percentage of participants

SECONDARY outcome

Timeframe: Day -10 to -1 (screening), Weeks 4 and 8

Population: Safety population

The potential ototoxic effects (hearing loss) of tobramycin were investigated by performing audiometric tests. Participants with a loss of auditory acuity greater than the 20 decibels auditory threshold are reported.

Outcome measures

Outcome measures
Measure
Bramitob
n=156 Participants
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=168 Participants
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Participants With a Hearing Threshold >20 Decibel in at Least One Ear
Screening
0 percentage of participants
0 percentage of participants
Participants With a Hearing Threshold >20 Decibel in at Least One Ear
Week 4
1.9 percentage of participants
1.2 percentage of participants
Participants With a Hearing Threshold >20 Decibel in at Least One Ear
Week 8
1.3 percentage of participants
1.2 percentage of participants

Adverse Events

Bramitob

Serious events: 6 serious events
Other events: 10 other events
Deaths: 0 deaths

TOBI

Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Bramitob
n=156 participants at risk
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=168 participants at risk
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Infections and infestations
Appendicitis
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.00%
0/168 • Day 0 to Week 8
Infections and infestations
Bronchitis
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.60%
1/168 • Number of events 1 • Day 0 to Week 8
Infections and infestations
Laryngitis
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.00%
0/168 • Day 0 to Week 8
Infections and infestations
Lung infection
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.00%
0/168 • Day 0 to Week 8
Congenital, familial and genetic disorders
Cystic fibrosis lung
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.60%
1/168 • Number of events 1 • Day 0 to Week 8
Injury, poisoning and procedural complications
Head injury
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.00%
0/168 • Day 0 to Week 8
Nervous system disorders
Syncope
0.64%
1/156 • Number of events 1 • Day 0 to Week 8
0.00%
0/168 • Day 0 to Week 8

Other adverse events

Other adverse events
Measure
Bramitob
n=156 participants at risk
tobramycin / Bramitob administered 300mg twice a day for 4 weeks
TOBI
n=168 participants at risk
tobramycin / TOBI administered 300mg twice a day for 4 weeks
Respiratory, thoracic and mediastinal disorders
Cough
6.4%
10/156 • Number of events 12 • Day 0 to Week 8
6.0%
10/168 • Number of events 11 • Day 0 to Week 8

Additional Information

Chiesi Clinical Trials

Chiesi Farmaceutici S.p.A.

Results disclosure agreements

  • Principal investigator is a sponsor employee Upon PI submission of publication to Sponsor, Sponsor will complete its review within 90 days after receipt. At Sponsor's request, publication may be delayed up to 120 additional days for Sponsor to file necessary patent applications. In multicenter trials, PIs will postpone single center publications until after multicenter data publication. If no multicenter publication occurs within 12 months of trial completion, PI may publish single center results, subject to review timelines listed above.
  • Publication restrictions are in place

Restriction type: OTHER