Resumen

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.

Elegibilidad

Edad mínima: 6 YearsSexo: ALL

Criterios de inclusión

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 \< 90% predicted
Be able to perform all the techniques necessary to measure lung function

Criterios de exclusión

Investigators, site personnel directly affiliated with this study, 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 nebulized hypertonic saline in the 4 weeks prior to visit 1
Have had a significant episode of hemoptysis (\>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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Ubicaciones

Hospital Interzonal Dr Jose Penna Bahia Blanca

Bahía Blanca, Buenos Aires, Argentina

Hospital de Ninos Dr Ricardo Gutierrez, Pediatria

CABA, Buenos Aires, Argentina

Hospital Gral. de Ninos Pedro de Elizalde

Ciudad Autonoma, Buenos Aires, Argentina

Hospital de Ninos Superiora Sor Maria Ludovica de La Plata

La Plata, Buenos Aires, Argentina

Hospital Pediatrico Dr Humberto J Notti

Villa Nueva, Mendoza Province, Argentina

Atención Integral en Reumatologia (AIR)

Buenos Aires, Argentina

Clinica Universitaria Privada Reina Fabiola - Universidad Cotolica de Cordoba

Córdoba, Argentina

Hospital de Ninos del la Santisima Trinidad