This trial is active, not recruiting.

Condition cystic fibrosis
Treatments inhaled mannitol, placebo comparator: arm b - control
Phase phase 3
Sponsor Pharmaxis
Start date October 2014
End date February 2017
Trial size 440 participants
Trial identifier NCT02134353, DPM-CF-303


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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Active treatment. Inhaled Mannitol
inhaled mannitol
Inhaled mannitol 400 mg BD for 26 weeks
(Placebo Comparator)
Arm B
placebo comparator: arm b - control
Placebo Comparator: Arm B - Control BD for 26 weeks

Primary Outcomes

Mean change in FEV1 (mL) from baseline (Visit 1) over the 26-week treatment period (to Visit 4).
time frame: 26 weeks

Secondary Outcomes

Mean change from baseline FVC (mL) over the 26-week treatment period
time frame: 26 weeks

Eligibility Criteria

Male or female participants from 18 years up to 99 years old.

Inclusion Criteria: 1. Have given written informed consent to participate in this trial in accordance with local regulations; 2. 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); 3. Be aged at least 18 years old; 4. Have FEV1 > 40 % and < 90% predicted (using NHanes III [1]); 5. Be able to perform all the techniques necessary to measure lung function; 6. Be adherent with maintenance therapies (antibiotics and or rhDNase), if used, for at least 80% of the time in the two weeks prior to visit 1 and 7. If rhDNase and/or maintenance antibiotic are being used treatment must have been established at least 1 month prior to screening (Visit 0). The subject should remain on the rhDNase and / or maintenance antibiotics for the duration of the trial. The subject should not commence treatment with rhDNase or maintenance antibiotics during the trial Exclusion Criteria: 1. Be investigators, site personnel directly affiliated with this trial, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted; 2. Be considered "terminally ill" or eligible for lung transplantation; 3. Have had a lung transplant; 4. Be using maintenance nebulized hypertonic saline in the 2 weeks prior to visit 1; 5. Have had a significant episode of hemoptysis (> 60 mL) in the three months prior to Visit 0; 6. Have had a myocardial infarction in the three months prior to Visit 0; 7. Have had a cerebral vascular accident in the three months prior to Visit 0; 8. Have had major ocular surgery in the three months prior to Visit 0; 9. Have had major abdominal, chest or brain surgery in the three months prior to Visit 0; 10. Have a known cerebral, aortic or abdominal aneurysm; 11. Be breast feeding or pregnant, or plan to become pregnant while in the trial; 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only); 13. Be participating in another investigative drug trial, parallel to, or within 4 weeks of screening (Visit 0); 14. Have a known allergy to mannitol; 15. Be using non-selective oral beta blockers; 16. Have uncontrolled hypertension -i.e. systolic BP > 190 and / or diastolic BP > 100; 17. 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 18. Have a failed or incomplete MTT at trial entry (as evaluated in Section 19. The subject must not commence treatment with rhDNase or maintenance antibiotics during the trial.

Additional Information

Official title Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy Trial in Adult Cystic Fibrosis Subjects
Principal investigator Moira Aitken, MD
Description This is a double-blind, randomized, parallel arm, controlled, multicenter, and interventional clinical trial. Potential subjects will sign the informed consent form (ICF) and be assessed for eligibility. After satisfying all inclusion & exclusion criteria, subjects will be given a mannitol tolerance test (MTT). Those subjects that pass the MTT will be randomized to receive inhaled mannitol (400 mg b.i.d.) or control b.i.d. for a period of 26-weeks.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Pharmaxis.