Trial Outcomes & Findings for Open Label Extension to Bridging Study CTBM100C2303 (NCT NCT00982930)
NCT ID: NCT00982930
Last Updated: 2021-06-02
Results Overview
AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product.
COMPLETED
PHASE3
55 participants
From first administration of study drug to study completion (up to approximately 25 weeks)
2021-06-02
Participant Flow
Participants who had completed all visits in the core study CTBM100C2303 took part in this extension study at 16 centers in 8 countries Bulgaria, Egypt, Estonia, Latvia, India, Lithuania, Romania and Russia from 12 August 2009 to 6 October 2011.
A total of 55 participants who completed all visits in the core study CTBM100C2303 and received one cycle (Cycle 1) of either Tobramycin Inhalation Powder (TIP) or matching placebo entered into the extension study and received 3 additional TIP cycles (Cycles 2, 3 and 4).
Participant milestones
| Measure |
Tobramycin Inhalation Powder (TIP)
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, twice a day (b.i.d), given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Overall Study
STARTED
|
55
|
|
Overall Study
COMPLETED
|
51
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Tobramycin Inhalation Powder (TIP)
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, twice a day (b.i.d), given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Administrative Problems
|
2
|
Baseline Characteristics
Number analyzed were the number of participants with data available for analyses at given time point.
Baseline characteristics by cohort
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Age, Continuous
|
12.9 years
STANDARD_DEVIATION 4.44 • n=55 Participants
|
|
Sex: Female, Male
Female
|
35 Participants
n=55 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=55 Participants
|
|
Baseline Forced Expiratory Volume in One Second (FEV1) Percent Predicted
|
59.9 percentage
STANDARD_DEVIATION 16.24 • n=52 Participants • Number analyzed were the number of participants with data available for analyses at given time point.
|
|
Baseline Forced Vital Capacity (FVC) Percent Predicted
|
75.2 percentage
STANDARD_DEVIATION 17.05 • n=52 Participants • Number analyzed were the number of participants with data available for analyses at given time point.
|
|
Baseline Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted
|
37.2 percentage
STANDARD_DEVIATION 20.19 • n=52 Participants • Number analyzed were the number of participants with data available for analyses at given time point.
|
|
Baseline P. aeruginosa Sputum Density
|
7.3 log10 Colony Forming Units (CFU)
STANDARD_DEVIATION 1.81 • n=50 Participants • Number analyzed were the number of participants with data available for analyses at given time point.
|
|
Baseline P. aeruginosa Tobramycin Minimal Inhibitory Concentration (MIC)
|
4.6 microgram/ mL (µg/mL)
STANDARD_DEVIATION 14.60 • n=55 Participants
|
PRIMARY outcome
Timeframe: From first administration of study drug to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Participants With Adverse Events (AEs)
|
47.3 percentage of participants
|
PRIMARY outcome
Timeframe: From time of consent to 4 weeks after study completion (up to approximately 29 weeks)Population: Safety Population included all enrolled participants who received at least one dose of study drug.
SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Participants With Serious Adverse Events (SAEs)
|
5.5 percentage of participants
|
PRIMARY outcome
Timeframe: From time of consent to 4 weeks after study completion (up to approximately 29 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Death Cases
|
0 percentage of participants
|
PRIMARY outcome
Timeframe: From first administration of study drug to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product. SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Participants With Adverse Events and Serious Adverse Events Leading to Permanent Study Discontinuation
Discontinuation Due to Serious Adverse Events
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events and Serious Adverse Events Leading to Permanent Study Discontinuation
Discontinuation Due to Adverse Events
|
1.8 percentage of participants
|
PRIMARY outcome
Timeframe: From baseline to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
Shift from baseline in hematology and biochemistry values to above upper/below lower limit of normal at any time post-baseline were reported. Baseline for safety analyses was defined as the last measurement prior to first dose of study drug in the core study CTBM100C2303. Change to low referred to number of participants with normal or high values at baseline. Change to high referred to number of participants with normal or low values at baseline.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Eosinophils - high
|
33.3 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Lymphocytes - high
|
32.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Neutrophils (Seg. + Bands) - high
|
35.7 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Basophils - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Eosinophils - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Eosinophils - high
|
37.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Hematocrit - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Hematocrit - high
|
29.5 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Hemoglobin - low
|
5.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Hemoglobin - high
|
6.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Lymphocytes - low
|
12.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Lymphocytes - high
|
39.5 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Monocytes - low
|
14.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Platelet count (direct) - high
|
24.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Alkaline phosphatase, serum - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Alkaline phosphatase, serum - high
|
22.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Blood Urea Nitrogen (BUN) - low
|
16.7 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Blood Urea Nitrogen (BUN) - high
|
10.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Calcium -low
|
12.7 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Creatinine - low
|
36.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Glucose - high
|
15.1 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Phosphate (Inorganic Phosphorus) - low
|
1.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Potassium - high
|
7.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Serum bicarbonate - high
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Sodium - low
|
10.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hematology (Hem): Absolute Basophils - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Basophils - high
|
16.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Eosinophils - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Lymphocytes - low
|
10.6 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Monocytes - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Monocytes - high
|
20.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Absolute Neutrophils (Seg. + Bands) - low
|
28.3 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Basophils - high
|
70.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Monocytes - high
|
42.5 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Neutrophils (Seg. + Bands) - low
|
37.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Neutrophils (Seg. + Bands) - high
|
10.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: Platelet count (direct) - low
|
2.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: RBC- low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: RBC- high
|
27.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: WBC (total)- low
|
20.5 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Hem: WBC (total)- high
|
31.7 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Biochemistry (Bio): Albumin - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Albumin - high
|
32.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Bilirubin (direct/conjugated) -low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Bilirubin (direct/conjugated) -high
|
11.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Bilirubin (total) - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Bilirubin (total) - high
|
7.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Calcium - high
|
11.1 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Chloride - low
|
9.1 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Chloride - high
|
1.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Creatinine - high
|
1.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Gamma Glutamyltransferase - low
|
1.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Gamma Glutamyltransferase - high
|
10.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Glucose - low
|
24.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Phosphate (Inorganic Phosphorus) - high
|
47.1 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Potassium - low
|
3.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: SGOT (AST) - low
|
3.7 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: SGOT (AST) - high
|
30.4 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: SGPT (ALT) - low
|
0.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: SGPT (ALT) - high
|
39.0 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Serum bicarbonate - low
|
45.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Sodium - high
|
5.5 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Total Protein (Serum) - low
|
1.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Total Protein (Serum) - high
|
27.9 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Uric Acid - low
|
1.8 percentage of participants at risk
|
|
Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
Bio: Uric Acid - high
|
16.0 percentage of participants at risk
|
PRIMARY outcome
Timeframe: Pre-dose and 30 minutes Post-dose on Day 1 and Day 29 of every Cycle (2, 3, 4)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
Airway Reactivity was defined as ≥20% FEV1 relative decrease in percent predicted from pre dose to 30 minutes post dose. FEV1 was defined as the volume of air expired in 1 second. 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 = 100\* (30 minutes post dose - pre dose) / pre dose assessed by number and percentage of participants with a decrease in ≥20% FEV1 percent predicted from pre dose to 30 minutes post dose. Baseline for was defined as the last measurement prior to first dose of study drug in the core study CTBM100C2303.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 2: Day 1
|
0.0 percentage change
Standard Deviation 12.07
|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 2: Day 29
|
-3.0 percentage change
Standard Deviation 7.21
|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 3: Day 1
|
-1.4 percentage change
Standard Deviation 13.33
|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 3: Day 29
|
-1.8 percentage change
Standard Deviation 6.67
|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 4: Day 1
|
-3.0 percentage change
Standard Deviation 11.01
|
|
Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Cycle 4: Day 29
|
-0.6 percentage change
Standard Deviation 6.82
|
PRIMARY outcome
Timeframe: From first dose of study drug to study completion (up to approximately 25 weeks)Population: Safety Population (Audiology Subgroup) included all participants in the safety population with at least one audiology testing. Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with normal hearing at baseline with data available for analyses at given time point.
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
n=22 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 1; >= 20 dB Decrease in at Least One Frequency In Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 29; ˃= 10 dB decrease in 3 Consecutive Frequencies in Either Ear
|
5.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 29; >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 3: Day 29; >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
|
4.5 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 3: Day 29; >= 20 dB Decrease in at Least One Frequency In Either Ear
|
4.5 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 1; ˃= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
|
5.6 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 1: >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 2: Day 29; >= 20 dB Decrease in at Least One Frequency In Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 3: Day 29; ˃= 10 dB Decrease in 3 Consecutive Frequencies in Either Ear
|
9.1 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 4: Day 29; ˃= 10 dB decrease in 3 Consecutive Frequencies in Either Ear
|
4.5 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 4: Day 29; >= 15 dB Decrease In 2 Consecutive Frequencies in Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycle 4: Day 29; >= 20 dB Decrease in at Least One Frequency In Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Day 57; ˃= 10 dB decrease in 3 Consecutive Frequencies in Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Day 57: >= 15 dB Decrease in 2 Consecutive Frequencies in Either Ear
|
0.0 percentage of participants
|
|
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Follow Up: Day 57; >= 20 dB Decrease in at Least One Frequency In Either Ear
|
0.0 percentage of participants
|
PRIMARY outcome
Timeframe: From first administration of study drug to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product. SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Mild, No
|
13 Participants
|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Moderate, No
|
9 Participants
|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Mild, Yes
|
1 Participants
|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Moderate, Yes
|
3 Participants
|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Severe, Yes
|
0 Participants
|
|
Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
Severe, No
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
FEV1 was defined as the volume of air expired in 1 second. 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 = 100\* (30 minutes post dose - pre dose) / pre dose assessed by number and percentage of participants with a decrease in ≥20% FEV1 percent predicted from pre dose to 30 minutes post dose. Baseline for was defined as the last measurement prior to first dose of study drug in the core study CTBM100C2303. Termination referred to the last available pre dose post-baseline measurement.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 2: Day 29
|
13.5 percentage change
Standard Deviation 23.42
|
|
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
Follow Up: Day 57
|
13.5 percentage change
Standard Deviation 20.62
|
|
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
Termination
|
13.9 percentage change
Standard Deviation 21.86
|
|
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 3: Day 29
|
14.8 percentage change
Standard Deviation 22.40
|
|
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 4: Day 29
|
17.3 percentage change
Standard Deviation 23.56
|
SECONDARY outcome
Timeframe: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
Relative change from baseline in FVC % predicted to end of dosing in each cycle and study completion were reported. Relative change from baseline was defined as: Relative change = 100\* (Post baseline- baseline) / baseline. Baseline was defined as last measurement prior to first dose of study drug in the core study CTBM100C2303. Termination referred to the last available pre dose post-baseline measurement.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 2: Day 29
|
7.0 percentage change
Standard Deviation 17.46
|
|
Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 3: Day 29
|
7.2 percentage change
Standard Deviation 16.89
|
|
Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 4: Day 29
|
9.6 percentage change
Standard Deviation 16.87
|
|
Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
Follow Up: Day 57
|
7.4 percentage change
Standard Deviation 14.99
|
|
Change From Baseline in Forced Vital Capacity (FVC) Percent (%) Predicted to End of Dosing at Each Cycle and Study Completion
Termination
|
7.9 percentage change
Standard Deviation 15.24
|
SECONDARY outcome
Timeframe: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
Relative change from baseline in FEF25-75% predicted to end of dosing in each cycle and study completion were reported. Relative change from baseline was defined as: Relative change = 100\* (Post baseline- baseline) / baseline. Baseline was defined as last measurement prior to first dose of study drug in the core study CTBM100C2303. Termination referred to the last available pre dose post-baseline measurement.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 2: Day 29
|
35.2 percentage change
Standard Deviation 55.86
|
|
Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
Follow Up: Day 57
|
35.9 percentage change
Standard Deviation 58.08
|
|
Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
Termination
|
34.7 percentage change
Standard Deviation 57.98
|
|
Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 3: Day 29
|
44.7 percentage change
Standard Deviation 58.17
|
|
Change From Baseline in Forced Expiratory Flow Rate Over 25 Percent and 75 Percent (FEF25-75%) Predicted to End of Dosing at Each Cycle and Study Completion
Cycle 4; Day 29
|
40.5 percentage change
Standard Deviation 59.21
|
SECONDARY outcome
Timeframe: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
P. aeruginosa sputum density referred to overall density, defined as the sum of biotypes (mucoid, dry and small colony variant). If sub-isolates existed for CFU biotype mucoid or dry, then the sum of sub-isolates was analyzed. Absolute change from baseline was defined as: Absolute change = Post Baseline - Baseline. Baseline was defined as last measurement prior to first dose of study drug in the core study CTBM100C2303. Termination referred to the last available pre dose post-baseline measurement.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
Cycle 2: Day 29
|
-3.7 log10 Colony Forming Units (CFU)
Standard Deviation 2.76
|
|
Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
Cycle 3: Day 29
|
-3.5 log10 Colony Forming Units (CFU)
Standard Deviation 3.04
|
|
Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
Cycle 4: Day 29
|
-3.9 log10 Colony Forming Units (CFU)
Standard Deviation 2.82
|
|
Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
Follow Up: Day 57
|
-1.1 log10 Colony Forming Units (CFU)
Standard Deviation 2.82
|
|
Absolute Change From Baseline in Sputum Pseudomonas Aeruginosa Density [log10 Colony Forming Units (CFU) Per Gram Sputum] to End of Dosing at Each Cycle and Study Completion
Termination
|
-2.9 log10 Colony Forming Units (CFU)
Standard Deviation 3.16
|
SECONDARY outcome
Timeframe: Baseline, Day 29 of Cycle 2, 3, 4, Day 57 of Follow Up and Termination (Study Completion)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study. Number analyzed were the number of participants with data available for analyses at given time point.
Change in tobramycin MIC values for P. aeruginosa were reported for specimens and were used to assess the change in pathogen susceptibility to tobramycin before (baseline) and after (post-baseline) the treatment. Maximum MIC values from all biotypes were used. Change from baseline was defined as: Change = Post-baseline - Baseline. Baseline was defined as last measurement prior to first dose of study drug in the core studyCTBM100C2303.Termination referred to the last available pre dose post-baseline measurement.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
Cycle 3: Day 29
|
30.7 μg/mL
Standard Deviation 112.32
|
|
Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
Cycle 4: Day 29
|
24.0 μg/mL
Standard Deviation 90.13
|
|
Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
Follow Up: Day 57
|
28.6 μg/mL
Standard Deviation 111.71
|
|
Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
Termination
|
22.3 μg/mL
Standard Deviation 101.38
|
|
Change From Baseline in Pseudomonas Aeruginosa Minimum Inhibitory Concentration (MIC) to End of Dosing at Each Cycle and Study Completion
Cycle 2: Day 29
|
15.1 μg/mL
Standard Deviation 82.45
|
SECONDARY outcome
Timeframe: From first administration of study drug to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Percentage of Participants With Anti-Pseudomonal Antibiotic Use During The Treatment Period
|
9.1 percentage of participants
|
SECONDARY outcome
Timeframe: From first administration of study drug to study completion (up to approximately 25 weeks)Population: Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect. For calculation of days in hospitalization due to respiratory events, the end date was defined as the discharge date (if provided and even if after the end of the extension study), and otherwise as the date of last visit.
Outcome measures
| Measure |
Tobramycin Inhalation Powder
n=55 Participants
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Number of Days of Hospitalization Due to Respiratory Serious Adverse Events
|
13.0 days
Interval 8.0 to 18.0
|
Adverse Events
Tobramycin Inhalation Powder
Serious adverse events
| Measure |
Tobramycin Inhalation Powder
n=55 participants at risk
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Infections and infestations
Aspergillosis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Lung infection
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Pneumonia
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
Other adverse events
| Measure |
Tobramycin Inhalation Powder
n=55 participants at risk
Participants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, b.i.d., given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
|
|---|---|
|
Ear and labyrinth disorders
Hypoacusis
|
5.5%
3/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Gastrointestinal disorders
Abdominal pain
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Gastrointestinal disorders
Diarrhoea
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
General disorders
Pyrexia
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Ascariasis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Bronchitis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Bronchopulmonary aspergillosis
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Gastrointestinal candidiasis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Nasopharyngitis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Respiratory tract infection
|
9.1%
5/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Respiratory tract infection viral
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Sinusitis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Stenotrophomonas infection
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Upper respiratory tract infection
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Infections and infestations
Viral rhinitis
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Injury, poisoning and procedural complications
Vaccination complication
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Investigations
Blood calcium decreased
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Investigations
Hepatic enzyme increased
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Investigations
Protein urine
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Investigations
Protein urine present
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Investigations
White blood cell count increased
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Nervous system disorders
Headache
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
5/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
5.5%
3/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus polyp
|
3.6%
2/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.8%
1/55 • From first administration of study drug to study completion (up to approximately 25 weeks)
Safety Population included all enrolled participants who completed their participation in the core study (CTBM100C2303) and received at least one dose of study drug in the extension study.
|
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