Phase 3
Completed N=318
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
Source: ClinicalTrials.gov NCT00630812 ↗Enrolled (actual)
318
Serious AEs
20.0%
Results posted
Oct 2020
Primary outcomePrimary: Change in Absolute FEV1 From Baseline Over 26 Weeks — 106.53; 52.38 mL — p=0.059
◆ Published Evidence
Highly cited
121citations · ~9 / year
Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study.
Summary
The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies have demonstrated improvements in lung function, mucociliary clearance, changes in physical properties of mucus, 24 hour sputum weight and quality of life. The results of this study are to further investigate and confirm these findings in addition to examine the effect on antibiotic use and chest infections. It is hypothesised that inhaled mannitol will have beneficial effects compared to a control treatment. An open label phase of 26 weeks duration will follow the blinded 26 week phase. During the open label phase all subjects will receive active treatment.
Linked Publications (2)
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Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study.
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Inhaled mannitol for cystic fibrosis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Absolute FEV1 From Baseline Over 26 Weeks |
106.53; 52.38 | 0.059 |
| SECONDARY Change in FEV1 From Baseline Over 26 Weeks - Dornase Users |
78.60; 35.11 | 0.177 |
| SECONDARY Rate of Protocol Defined Pulmonary Exacerbations (PDPE) |
21; 18; 6; 4; 1; 1 | 0.520 |
| SECONDARY Hospitalisations Associated With Protocol Defined Pulmonary Exacerbations (PDPEs) |
162; 102; 18; 14; 3; 5 | 0.328 |
| SECONDARY Antibiotic Use Associated With PDPEs |
156; 98; 22; 19; 5; 2 | 0.368 |
| SECONDARY Absolute Change in FEV1 Percent Predicted at 26 Weeks |
3.14; 0.72 | 0.024 sig |
| SECONDARY Change in FVC (mL) Across 26 Weeks |
136.33; 64.98 | 0.022 sig |
| SECONDARY Change From Baseline FEF25-75 (mL/s) Over 26 Weeks |
84.65; 50.31 | 0.49 |
| SECONDARY Sputum Weight at Baseline in Response to First Dose of Treatment |
4.9; 3.5 | 0.042 sig |
Eligibility Criteria
Inclusion Criteria
- Have given written informed consent to participate in this study in accordance with local regulations
- Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype)
- Be aged > 6 years old
- Have FEV1 >40 % and 60 mL) in the three months prior to enrolment
- Have had a myocardial infarction in the three months prior to enrolment
- Have had a cerebral vascular accident in the three months prior to enrolment
- Have had major ocular surgery in the three months prior to enrolment
- Have had major abdominal, chest or brain surgery in the three months prior to enrolment
- Have a known cerebral, aortic or abdominal aneurysm
- Be breast feeding or pregnant, or plan to become pregnant while in the study
- Be using an unreliable form of contraception (female subjects at risk of pregnancy only)
- Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0
- Have a known allergy to mannitol
- Be using beta blockers
- Have uncontrolled hypertension - systolic BP > 190 and / or diastolic BP > 100
- Have a condition or be in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient's participation in the study
- Be 'Mannitol Tolerance Test positive'
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Data sourced from ClinicalTrials.gov (NCT00630812) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.