Phase 3
Completed N=423
A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects
Source: ClinicalTrials.gov NCT02134353 ↗Enrolled (actual)
423
Serious AEs
14.3%
Results posted
Sep 2020
Primary outcomePrimary: Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4). — 63; 8 mL — p=0.020
◆ Published Evidence
Emerging
18citations · ~4 / year
Efficacy and safety of inhaled dry-powder mannitol in adults with cystic fibrosis: An international, randomized controlled study.
Summary
This trial aims to provide prospective evidence of the safety and efficacy of mannitol 400 mg b.i.d. in subjects aged 18 years and above.
We hypothesize that inhaled mannitol 400 mg b.i.d. will increase the mean change from baseline FEV1 (mL) compared to control over the 26-week treatment period in adult subjects with cystic fibrosis. Any improvement in FEV1 is considered clinically meaningful, however, this trial has set a threshold of 80 mL for the purposes of determining an appropriate sample size for statistical power while retaining trial feasibility in an orphan disease population
Linked Publications
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Efficacy and safety of inhaled dry-powder mannitol in adults with cystic fibrosis: An international, randomized controlled study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4). |
63; 8 | 0.020 sig |
| SECONDARY Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period |
28; -12 | 0.128 |
Eligibility Criteria
Inclusion Criteria
- Have given written informed consent to participate in this trial 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 at least 18 years old;
- Have FEV1 > 40 % and 60 mL) in the three months prior to Visit 0;
- Have had a myocardial infarction in the three months prior to Visit 0;
- Have had a cerebral vascular accident in the three months prior to Visit 0;
- Have had major ocular surgery in the three months prior to Visit 0;
- Have had major abdominal, chest or brain surgery in the three months prior to Visit 0;
- Have a known cerebral, aortic or abdominal aneurysm;
- Be breast feeding or pregnant, or plan to become pregnant while in the trial;
- Be using an unreliable form of contraception (female subjects at risk of pregnancy only);
- Be participating in another investigative drug trial, parallel to, or within 4 weeks of screening (Visit 0);
- Have a known allergy to mannitol;
- Be using non-selective oral beta blockers;
- Have uncontrolled hypertension -i.e. 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 subject's participation in the trial;or
- Have a failed or incomplete MTT at trial entry (as evaluated in Section 8.1.1.1).
- The subject must not commence treatment with rhDNase or maintenance antibiotics during the trial.
Data sourced from ClinicalTrials.gov (NCT02134353) 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.