Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study

Syntara

Status and phase

Completed

Phase 3

Conditions

Cystic Fibrosis

Treatments

Drug: Mannitol

Drug: placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00446680

DPM-CF-301

Details and patient eligibility

About

The purpose of this study is to determine the efficacy and safety of chronic treatment with inhaled dry powder mannitol in subjects with cystic fibrosis. Previous studies have demonstrated an improvement in lung function related to small airways obstruction and a significant improvement in respiratory symptoms and quality of life after a 2 week treatment with mannitol. This current study seeks to support these early findings and to extend the evidence to support its use as a mucoactive therapy in cystic fibrosis. In particular, the hypothesis that enhanced mucus clearance will improve the lung function and clinical presentation in this population, will be investigated. We also hypothesize that enhanced mucociliary clearance will result in a sustained reduction in mucus load, thus providing less opportunity for bacteria to proliferate, affording a reduction in antibiotic use and hospitalizations. The initial 6 month blinded phase will be followed with an additional 6 months of open label treatment.

Enrollment

340 estimated patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Main Inclusion Criteria:

Written informed consent
Confirmed diagnosis of cystic fibrosis
Aged > 6 years
FEV1 >30 % and < 90% predicted
Able to perform all the techniques necessary to measure lung function

Main Exclusion Criteria:

"Terminally ill" or listed for lung transplantation
Had a lung transplant
Using nebulised hypertonic saline
Significant episode of haemoptysis (>60 mL) in the three months prior to enrolment
Recent myocardial infarction or cerebral vascular accident
Breast feeding or pregnant, or plan to become pregnant while in the study participating in another investigative drug study, parallel to, or within 4 weeks of study entry
Allergy or intolerance to mannitol
Using beta blockers
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