Effect of Hypertonic Saline on Mucus Clearance in Children Ages 5-12 With Cystic Fibrosis

• Status: Completed
• Trial end date: 2012-07-01
• Target enrollment: 0
• Participant gender: All

Summary

Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in adults with cystic fibrosis (CF). It is unclear, however, whether HS will benefit young patients suffering from CF. The investigators propose to further support the concept that HS can benefit children with mild CF lung disease by performing a relatively short, placebo controlled study of HS in 5-12 year olds, using lung function and mucociliary clearance as key outcome measures.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of North Carolina, Chapel Hill
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)

Criteria

Inclusion Criteria:

• Gender: Females or Males. If the subject is female and of childbearing potential (first menses has occurred), she must have a documented negative pregnancy test at screening and prior to each mucociliary clearance study. Those of childbearing potential must be abstinent or using an acceptable method of birth control (i.e. an Intrauterine Contraceptive Device with a failure rate of <1%, hormonal contraceptives or a barrier method).

• Age: 5-12 years, inclusive

• Diagnosis: Cystic fibrosis documented by a compatible clinical presentation and sweat chloride > 60 mEq/l or 2 disease causing CFTR mutations.

• Severity of the Disease: Suitable patients will have mild lung disease, as defined by:

• Pulmonary Function: Each patient must have an FEV1 of greater than or equal to 60% of predicted at the screening visit.

• Hemoglobin saturation: Patients must have an oxygen saturation of >92% on room air as determined by pulse oximetry at the screening visit.

• Informed consent - The patient and a parent or legally authorized guardian must agree to the subject's participation in the study by signing and dating the informed consent/assent forms after the nature of the study has been fully explained and all questions have been satisfactorily answered.

Exclusion Criteria:

• Unstable or asthmatic lung disease: As defined by a change in medical regimen during the preceding 2 weeks; an FEV1 15% below recent (within 6 months) clinical measurements. Patients with a history of co-existent asthma, as manifested by wheezing and significant bronchoreactivity (>15% increase in FEV1 with bronchodilator), will also be excluded.

• Other medication usage: Patients unable or unwilling to be withdrawn from hypertonic saline therapy for two weeks prior to Visit 1 (baseline MCC visit). Patients using Pulmozyme will be permitted to participate in this trial. Patients on chronic, cycling antibiotics will be required to have completed at least 2 full cycles of the prescribed antibiotic prior to enrollment and should not cycle on or off this therapy during the treatment period of the study.

• Spirometry Performance: Those subjects who are unable to perform acceptable, reproducible spirometry will be excluded from this study.

• Drug allergy: A history of allergy or intolerance to any of the study medications, including albuterol or hypertonic saline.

• Have received an investigational drug or therapy during the preceding 30 days.

• Have had radiation exposure within the past year that would cause them to exceed Federal Regulations by participating in this study.