Trial · NCT02134353

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

NCT02134353, Phase 3, Completed, Syntara; results first posted 2020-09-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects. Trials, record NCT02134353. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02134353

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects
NCT number: NCT02134353
Status: Completed
Phases: Phase 3
Enrollment: 423 participants, actual
Lead sponsor: Syntara
Conditions: Cystic Fibrosis
Interventions: Inhaled mannitol, Placebo Comparator: Arm B - Control
First posted: 2014-05-09
Last updated: 2020-10-28
Results first posted: 2020-09-03

Source ClinicalTrials.gov

Registered outcomes9

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4).	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).
Secondary	Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period	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).
Other	Time to First Pulmonary Exacerbation Over the 26-week Treatment Period	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
Other	Number of Days on Antibiotics (Oral, Inhaled or IV) Due to Pulmonary Exacerbation	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.
Other	Number of Days in Hospital Due to Pulmonary Exacerbation	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.
Other	Rate of Pulmonary Exacerbations Over the 26-week Treatment Period	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.
Other	The Incidence of Pulmonary Exacerbations	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.
Other	Mean Change From Baseline in Ease of Expectoration Measured Using a Visual Analogue Scale (VAS) Over 26 Weeks	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).
Other	Change From Baseline Over 26 Weeks in CFQ-R Respiratory Domain Score	26 weeks	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).

Source ClinicalTrials.gov

Outcomes10

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4).	26 weeks		mL	LEAST_SQUARES_MEAN
SECONDARY	Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period	26 weeks		mL	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Time to First Pulmonary Exacerbation Over the 26-week Treatment Period	26 weeks		days	MEDIAN
OTHER_PRE_SPECIFIED	Number of Days on Antibiotics (Oral, Inhaled or IV) Due to Pulmonary Exacerbation	26 weeks		days	MEAN
OTHER_PRE_SPECIFIED	Number of Days in Hospital Due to Pulmonary Exacerbation	26 weeks		Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	Rate of Pulmonary Exacerbations Over the 26-week Treatment Period	26 weeks		Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	The Incidence of Pulmonary Exacerbations	26 weeks		Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	Mean Change From Baseline in Ease of Expectoration Measured Using a Visual Analogue Scale (VAS) Over 26 Weeks	26 weeks		cm	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Change From Baseline Over 26 Weeks in CFQ-R Respiratory Domain Score	26 weeks	Intent to Treat (ITT) - All patients randomised.	score on a scale	LEAST_SQUARES_MEAN
POST_HOC	Absolute Change in Forced Expiratory Flow From 25% to 75% of Vital Capacity (FEF25-75) Over the 26-week Treatment Period.	26 weeks		mL/s	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group36

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4).	Experimental Arm A		63	95% Confidence Interval 25 to 100	mL
Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4).	Arm B - Control		8	95% Confidence Interval -27 to 44	mL
Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period	Experimental Arm A		28	95% Confidence Interval -14 to 70	mL
Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period	Arm B - Control		-12	95% Confidence Interval -53 to 29	mL
Time to First Pulmonary Exacerbation Over the 26-week Treatment Period	Experimental Arm A		NA	95% Confidence Interval NA to NA	days
Time to First Pulmonary Exacerbation Over the 26-week Treatment Period	Arm B - Control		NA	95% Confidence Interval NA to NA	days
Number of Days on Antibiotics (Oral, Inhaled or IV) Due to Pulmonary Exacerbation	Experimental Arm A		8.1	Standard Deviation 31.25	days
Number of Days on Antibiotics (Oral, Inhaled or IV) Due to Pulmonary Exacerbation	Arm B - Control		5.5	Standard Deviation 17.94	days
Number of Days in Hospital Due to Pulmonary Exacerbation	Experimental Arm A	0 days	192		Participants
Number of Days in Hospital Due to Pulmonary Exacerbation	Arm B - Control	0 days	199		Participants

1 to 10 of 36Source ClinicalTrials.gov

See all 36 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Experimental Arm A	209
Overall Study	STARTED	Arm B - Control	214
Overall Study	COMPLETED	Experimental Arm A	183
Overall Study	COMPLETED	Arm B - Control	190
Overall Study	NOT COMPLETED	Experimental Arm A	26
Overall Study	NOT COMPLETED	Arm B - Control	24

Source ClinicalTrials.gov

Adverse events70

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Condition Aggravated	General disorders	Experimental Arm A	56	207	84
other	Condition Aggravated	General disorders	Arm B - Control	59	213	75
other	Cough	Respiratory, thoracic and mediastinal disorders	Experimental Arm A	23	207	25
other	Cough	Respiratory, thoracic and mediastinal disorders	Arm B - Control	21	213	25
other	Haemoptysis	Respiratory, thoracic and mediastinal disorders	Experimental Arm A	20	207	27
other	Haemoptysis	Respiratory, thoracic and mediastinal disorders	Arm B - Control	20	213	33
other	Headache	Nervous system disorders	Experimental Arm A	12	207	41
other	Headache	Nervous system disorders	Arm B - Control	22	213	47
other	Upper Respiratory Tract Infection	Infections and infestations	Experimental Arm A	14	207	17
other	Upper Respiratory Tract Infection	Infections and infestations	Arm B - Control	11	213	12

1 to 10 of 70Source ClinicalTrials.gov

See all 70 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Experimental Arm A	0	207
Total, all-cause mortality	Arm B - Control	1	213
Total, other adverse events	Experimental Arm A	144	207
Total, other adverse events	Arm B - Control	140	213
Total, serious adverse events	Experimental Arm A	31	207
Total, serious adverse events	Arm B - Control	29	213

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33715994	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Experimental Arm A vs Arm B - Control: The Incidence of Pulmonary Exacerbations	Trial comparisons
Experimental Arm A vs Arm B - Control: deaths from any cause	Trial comparisons
Experimental Arm A vs Arm B - Control: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

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