Trial Outcomes & Findings for A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects (NCT NCT02134353)

NCT ID: NCT02134353

Last Updated: 2020-10-28

Results Overview

The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits. Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

423 participants

Primary outcome timeframe

26 weeks

Results posted on

2020-10-28

Participant Flow

Participant milestones

Participant milestones
Measure
Experimental Arm A
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg twice a day (BD) for 26 weeks
Arm B - Control
Arm B Control (mannitol 50mg) twice a day (BD) for 26 weeks
Overall Study
STARTED
209
214
Overall Study
COMPLETED
183
190
Overall Study
NOT COMPLETED
26
24

Reasons for withdrawal

Reasons for withdrawal
Measure
Experimental Arm A
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg twice a day (BD) for 26 weeks
Arm B - Control
Arm B Control (mannitol 50mg) twice a day (BD) for 26 weeks
Overall Study
Adverse Event
10
6
Overall Study
Lost to Follow-up
1
1
Overall Study
Lack of Efficacy
2
1
Overall Study
Withdrawal by Subject
12
13
Overall Study
Death
0
1
Overall Study
Pregnancy
0
1
Overall Study
Relocation
1
0
Overall Study
Other - Patient's Choice
0
1

Baseline Characteristics

A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Total
n=423 Participants
Total of all reporting groups
Region of Enrollment
New Zealand
2 participants
n=5 Participants
3 participants
n=7 Participants
5 participants
n=5 Participants
Age, Continuous
26.8 years
STANDARD_DEVIATION 7.63 • n=5 Participants
28.6 years
STANDARD_DEVIATION 10.75 • n=7 Participants
27.7 years
STANDARD_DEVIATION 9.37 • n=5 Participants
Sex: Female, Male
Female
92 Participants
n=5 Participants
107 Participants
n=7 Participants
199 Participants
n=5 Participants
Sex: Female, Male
Male
117 Participants
n=5 Participants
107 Participants
n=7 Participants
224 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
14 Participants
n=5 Participants
7 Participants
n=7 Participants
21 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
195 Participants
n=5 Participants
207 Participants
n=7 Participants
402 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
4 Participants
n=5 Participants
2 Participants
n=7 Participants
6 Participants
n=5 Participants
Race (NIH/OMB)
White
202 Participants
n=5 Participants
209 Participants
n=7 Participants
411 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
2 Participants
n=5 Participants
2 Participants
n=7 Participants
4 Participants
n=5 Participants
Region of Enrollment
Argentina
4 participants
n=5 Participants
2 participants
n=7 Participants
6 participants
n=5 Participants
Region of Enrollment
Romania
5 participants
n=5 Participants
5 participants
n=7 Participants
10 participants
n=5 Participants
Region of Enrollment
Hungary
9 participants
n=5 Participants
8 participants
n=7 Participants
17 participants
n=5 Participants
Region of Enrollment
United States
57 participants
n=5 Participants
59 participants
n=7 Participants
116 participants
n=5 Participants
Region of Enrollment
Czechia
0 participants
n=5 Participants
1 participants
n=7 Participants
1 participants
n=5 Participants
Region of Enrollment
Ukraine
31 participants
n=5 Participants
32 participants
n=7 Participants
63 participants
n=5 Participants
Region of Enrollment
Russia
21 participants
n=5 Participants
20 participants
n=7 Participants
41 participants
n=5 Participants
Region of Enrollment
Spain
6 participants
n=5 Participants
5 participants
n=7 Participants
11 participants
n=5 Participants
Region of Enrollment
Canada
7 participants
n=5 Participants
6 participants
n=7 Participants
13 participants
n=5 Participants
Region of Enrollment
Belgium
3 participants
n=5 Participants
3 participants
n=7 Participants
6 participants
n=5 Participants
Region of Enrollment
Poland
22 participants
n=5 Participants
22 participants
n=7 Participants
44 participants
n=5 Participants
Region of Enrollment
Italy
7 participants
n=5 Participants
8 participants
n=7 Participants
15 participants
n=5 Participants
Region of Enrollment
Mexico
3 participants
n=5 Participants
2 participants
n=7 Participants
5 participants
n=5 Participants
Region of Enrollment
South Africa
4 participants
n=5 Participants
6 participants
n=7 Participants
10 participants
n=5 Participants
Region of Enrollment
Slovakia
8 participants
n=5 Participants
9 participants
n=7 Participants
17 participants
n=5 Participants
Region of Enrollment
Israel
4 participants
n=5 Participants
7 participants
n=7 Participants
11 participants
n=5 Participants
Region of Enrollment
Australia
2 participants
n=5 Participants
2 participants
n=7 Participants
4 participants
n=5 Participants
Region of Enrollment
Bulgaria
6 participants
n=5 Participants
7 participants
n=7 Participants
13 participants
n=5 Participants
Region of Enrollment
Greece
4 participants
n=5 Participants
3 participants
n=7 Participants
7 participants
n=5 Participants
Region of Enrollment
Turkey
4 participants
n=5 Participants
4 participants
n=7 Participants
8 participants
n=5 Participants
%Predicted FEV1 at Screening
>80% to <=90%
31 Participants
n=5 Participants
27 Participants
n=7 Participants
58 Participants
n=5 Participants
%Predicted FEV1 at Screening
>70% to <=80%
54 Participants
n=5 Participants
52 Participants
n=7 Participants
106 Participants
n=5 Participants
%Predicted FEV1 at Screening
>40% to <=70%
124 Participants
n=5 Participants
135 Participants
n=7 Participants
259 Participants
n=5 Participants
CFTR Mutation
Both deltaF508
55 Participants
n=5 Participants
48 Participants
n=7 Participants
103 Participants
n=5 Participants
CFTR Mutation
One deltaF508
91 Participants
n=5 Participants
89 Participants
n=7 Participants
180 Participants
n=5 Participants
CFTR Mutation
At least one other known mutation
28 Participants
n=5 Participants
37 Participants
n=7 Participants
65 Participants
n=5 Participants
CFTR Mutation
Both unknown
35 Participants
n=5 Participants
40 Participants
n=7 Participants
75 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
0
121 Participants
n=5 Participants
135 Participants
n=7 Participants
256 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
1
57 Participants
n=5 Participants
43 Participants
n=7 Participants
100 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
2
20 Participants
n=5 Participants
23 Participants
n=7 Participants
43 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
3
11 Participants
n=5 Participants
9 Participants
n=7 Participants
20 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
4
0 Participants
n=5 Participants
3 Participants
n=7 Participants
3 Participants
n=5 Participants
Hospitalisations due to exacerbations in 12 months prior to screening
>4
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
0
109 Participants
n=5 Participants
120 Participants
n=7 Participants
229 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
1
58 Participants
n=5 Participants
47 Participants
n=7 Participants
105 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
2
28 Participants
n=5 Participants
32 Participants
n=7 Participants
60 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
3
13 Participants
n=5 Participants
11 Participants
n=7 Participants
24 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
4
1 Participants
n=5 Participants
3 Participants
n=7 Participants
4 Participants
n=5 Participants
Exacerbations treated with IV antibiotics in 12 months before screening
>4
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 26 weeks

The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits. Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4).
63 mL
Interval 25.0 to 100.0
8 mL
Interval -27.0 to 44.0

SECONDARY outcome

Timeframe: 26 weeks

To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control for improving lung function as measured by mean change from baseline forced vital capacity (FVC) (mL) over the 26-week treatment period in adult subjects with cystic fibrosis (CF). The mean absolute change from baseline FVC (mL) over 26 weeks will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the change from baseline averaged over the treatment period (ie the average of the changes at 6 weeks, 14 weeks and 26 weeks). Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period
28 mL
Interval -14.0 to 70.0
-12 mL
Interval -53.0 to 29.0

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control in increasing the time to first protocol defined pulmonary exacerbation over the 26-week treatment period in adult subjects with CF. Protocol defined pulmonary exacerbations are those where 4 or more symptoms are recorded and are treated with IV antibiotics

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Time to First Pulmonary Exacerbation Over the 26-week Treatment Period
NA days
Too few participants with protocol defined pulmonary exacerbations (PDPE), median time to first PDPE not reached
NA days
Too few participants with protocol defined pulmonary exacerbations (PDPE), median time to first PDPE not reached

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for decreasing the number of days on antibiotics due to protocol defined pulmonary exacerbations. Overlapping antibiotics are counted separately.

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Number of Days on Antibiotics (Oral, Inhaled or IV) Due to Pulmonary Exacerbation
8.1 days
Standard Deviation 31.25
5.5 days
Standard Deviation 17.94

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for decreasing the number of days in hospital due to protocol defined pulmonary exacerbation.

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Number of Days in Hospital Due to Pulmonary Exacerbation
0 days
192 Participants
199 Participants
Number of Days in Hospital Due to Pulmonary Exacerbation
1-7 days
1 Participants
4 Participants
Number of Days in Hospital Due to Pulmonary Exacerbation
8-14 days
4 Participants
6 Participants
Number of Days in Hospital Due to Pulmonary Exacerbation
15-21 days
5 Participants
3 Participants
Number of Days in Hospital Due to Pulmonary Exacerbation
22-28 days
3 Participants
2 Participants
Number of Days in Hospital Due to Pulmonary Exacerbation
>28 days
4 Participants
0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether inhaled mannitol (400 mg b.i.d.) decreases the rate of protocol defined pulmonary exacerbations over the 26-week treatment period compared to control in adult subjects with CF. Protocol defined pulmonary exacerbations defined by having 4 or more symptoms and treated with IV antibiotics.

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Rate of Pulmonary Exacerbations Over the 26-week Treatment Period
0 protocol defined pulm exac
181 Participants
185 Participants
Rate of Pulmonary Exacerbations Over the 26-week Treatment Period
1 protocol defined pulm exac
24 Participants
29 Participants
Rate of Pulmonary Exacerbations Over the 26-week Treatment Period
2 protocol defined pulm exac
3 Participants
0 Participants
Rate of Pulmonary Exacerbations Over the 26-week Treatment Period
>2 protocol defined pulm exac
1 Participants
0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for decreasing incidence of protocol defined pulmonary exacerbations, where incidence is defined as the proportion of subjects with 1 or more exacerbation during the 26 week period.

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
The Incidence of Pulmonary Exacerbations
28 Participants
29 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for improving ease of expectoration. The visual analogue scale (VAS) was 10cm. The position marked by the subject was converted into a score from 0 to 100 where 0 was the worst possible outcome and 100 was the best possible outcome. The VAS was completed at baseline, 6, 14 and 26 weeks. The mean absolute change from baseline over 26 weeks (ie the average of the changes at 6,14 and 26 weeks) is the outcome measure and will be compared between the two treatment groups with a REML based repeated measures approach. Least square means presented are for the change from baseline averaged over the treatment period (ie the average of the changes at 6 weeks, 14 weeks and 26 weeks). Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Mean Change From Baseline in Ease of Expectoration Measured Using a Visual Analogue Scale (VAS) Over 26 Weeks
0.795 cm
Interval 0.556 to 1.034
0.537 cm
Interval 0.306 to 0.767

OTHER_PRE_SPECIFIED outcome

Timeframe: 26 weeks

Population: Intent to Treat (ITT) - All patients randomised.

To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for improving respiratory symptoms measured by Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain. The CFQ-R respiratory domain score is a scale from 0 to 100. Higher scores are a more favourable response. The mean absolute change from baseline over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the change from baseline averaged over the treatment period (ie the average of the changes at 6 weeks, 14 weeks and 26 weeks). Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Change From Baseline Over 26 Weeks in CFQ-R Respiratory Domain Score
0.308 score on a scale
Interval -1.554 to 2.169
-0.562 score on a scale
Interval -2.357 to 1.234

POST_HOC outcome

Timeframe: 26 weeks

The mean absolute change from baseline FEF25-75 (mL/s) over weeks 6, 14 and 26 will be compared between the two treatment groups with a REML based repeated measures approach Least square means presented are for the change from baseline averaged over the treatment period (ie the average of the changes at 6 weeks, 14 weeks and 26 weeks). Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).

Outcome measures

Outcome measures
Measure
Experimental Arm A
n=209 Participants
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks
Arm B - Control
n=214 Participants
Arm B Control BD (mannitol 50mg) for 26 weeks
Absolute Change in Forced Expiratory Flow From 25% to 75% of Vital Capacity (FEF25-75) Over the 26-week Treatment Period.
109 mL/s
Interval 54.0 to 164.0
22 mL/s
Interval -32.0 to 75.0

Adverse Events

Experimental Arm A

Serious events: 31 serious events
Other events: 144 other events
Deaths: 0 deaths

Arm B - Control

Serious events: 29 serious events
Other events: 140 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
Experimental Arm A
n=207 participants at risk
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks Subjects randomised and treated.
Arm B - Control
n=213 participants at risk
Arm B Control BD (mannitol 50mg) for 26 weeks Subjects randomised and treated.
General disorders
Condition Aggravated
9.7%
20/207 • Number of events 25 • 26 weeks
7.0%
15/213 • Number of events 15 • 26 weeks
Infections and infestations
Lung Infection
0.48%
1/207 • Number of events 1 • 26 weeks
0.94%
2/213 • Number of events 2 • 26 weeks
Infections and infestations
Pneumonia
0.48%
1/207 • Number of events 1 • 26 weeks
0.94%
2/213 • Number of events 2 • 26 weeks
Infections and infestations
Lobar pneumonia
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Infections and infestations
Bronchitis
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Infections and infestations
Lower Respiratory Tract Infection
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Infections and infestations
Lung infection pseudomonal
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Infections and infestations
Meningitis bacterial
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Infections and infestations
Pneumonia bacterial
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Infections and infestations
Pneumonia viral
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Infections and infestations
Sepsis
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Infections and infestations
Staphylococcal bacteraemia
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Infections and infestations
Upper respiratory tract infection
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Haemoptysis
0.48%
1/207 • Number of events 1 • 26 weeks
1.4%
3/213 • Number of events 3 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Dyspnoea
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Pneumomediastinum
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Pneumothorax
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Gastrointestinal disorders
Pancreatitis acute
0.48%
1/207 • Number of events 1 • 26 weeks
0.94%
2/213 • Number of events 2 • 26 weeks
Gastrointestinal disorders
Abdominal pain upper
0.48%
1/207 • Number of events 1 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Gastrointestinal disorders
Distal intestinal obstruction syndrome
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Hepatobiliary disorders
Cholelithiasis
0.00%
0/207 • 26 weeks
0.94%
2/213 • Number of events 2 • 26 weeks
Psychiatric disorders
Depression
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Psychiatric disorders
Drug abuse
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Injury, poisoning and procedural complications
Intentional overdose
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Nervous system disorders
Guillain-Barre syndrome
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks
Renal and urinary disorders
Nephrolithiasis
0.48%
1/207 • Number of events 1 • 26 weeks
0.00%
0/213 • 26 weeks
Skin and subcutaneous tissue disorders
Subcutaneous emphysema
0.00%
0/207 • 26 weeks
0.47%
1/213 • Number of events 1 • 26 weeks

Other adverse events

Other adverse events
Measure
Experimental Arm A
n=207 participants at risk
Active treatment. Inhaled Mannitol Inhaled mannitol: Inhaled mannitol 400 mg BD for 26 weeks Subjects randomised and treated.
Arm B - Control
n=213 participants at risk
Arm B Control BD (mannitol 50mg) for 26 weeks Subjects randomised and treated.
General disorders
Condition Aggravated
27.1%
56/207 • Number of events 84 • 26 weeks
27.7%
59/213 • Number of events 75 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Cough
11.1%
23/207 • Number of events 25 • 26 weeks
9.9%
21/213 • Number of events 25 • 26 weeks
Respiratory, thoracic and mediastinal disorders
Haemoptysis
9.7%
20/207 • Number of events 27 • 26 weeks
9.4%
20/213 • Number of events 33 • 26 weeks
Nervous system disorders
Headache
5.8%
12/207 • Number of events 41 • 26 weeks
10.3%
22/213 • Number of events 47 • 26 weeks
Infections and infestations
Upper Respiratory Tract Infection
6.8%
14/207 • Number of events 17 • 26 weeks
5.2%
11/213 • Number of events 12 • 26 weeks
General disorders
Pyrexia
6.3%
13/207 • Number of events 18 • 26 weeks
3.8%
8/213 • Number of events 9 • 26 weeks
Infections and infestations
Nasopharyngitis
5.8%
12/207 • Number of events 17 • 26 weeks
4.7%
10/213 • Number of events 11 • 26 weeks

Additional Information

Dr Brett Charlton

Pharmaxis

Phone: +61 2 9454 7200

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place