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

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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

Key facts

Study ID: NCT02134353
Run by: Syntara
People needed: 423
Starts: 2014-09-01
Expected to finish: 2017-02-01
Last updated by the study team: 2020-10-28

Who can join

Age: 18 and older, up to 99. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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 < 90% predicted (using NHanes III [1]);
Be able to perform all the techniques necessary to measure lung function;
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
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

You may not qualify if…

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;
Be considered "terminally ill" or eligible for lung transplantation;
Have had a lung transplant;
Be using maintenance nebulized hypertonic saline in the 2 weeks prior to visit 1;
Have had a significant episode of hemoptysis (> 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.

Where it is running

University of Arizona — Tucson, Arizona, United States
University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States
Pediatric Pulmonology — Long Beach, California, United States
University of CA, Davis — Sacramento, California, United States
Hartford Hospital — Hartford, Connecticut, United States
Dr Mitchell Rothstein — Jacksonville, Florida, United States
University of Miami — Miami, Florida, United States
Central Florida Pulmonary Group, P.A. — Orlando, Florida, United States
Tampa General Hospital — Tampa, Florida, United States
Cystic Fibrosis Center of Chicago — Glenview, Illinois, United States
Loyola University Medical Center — Maywood, Illinois, United States
Illinois Lung Institute — Peoria, Illinois, United States
University of Kansas Hospital — Kansas City, Kansas, United States
Kosair Charities Pediatric Clinical Research Unit — Louisville, Kentucky, United States
John Hopkins — Baltimore, Maryland, United States
Massachusetts General Hospital — Boston, Massachusetts, United States
Spectrum Health Offices of Research Administration — Grand Rapids, Michigan, United States
University of Minnesota — Minneapolis, Minnesota, United States
University of Mississippi Medical Center — Jackson, Mississippi, United States
Dr Joseph Ojile — St Louis, Missouri, United States
Dartmouth-Hitchcock Specialty Care — Bedford, New Hampshire, United States
Long Island Jewish Medical Center — New Hyde Park, New York, United States
The Cystic Fibrosis Center Beth Israel Medical Center — New York, New York, United States
Dr Allen Dozor — Valhalla, New York, United States
Dr Lawrence Sinde — Mobile, Alabama, United States

Full record on ClinicalTrials.gov

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