Aerosolized Hypertonic Xylitol Versus Hypertonic Saline in Cystic Fibrosis (CF) Subjects

• Status: Completed
• Trial end date: 2018-04-09
• Target enrollment: 0
• Participant gender: All

Summary

Cystic fibrosis (CF) lung disease is characterized by chronic bacterial colonization and recurrent infection of the airways. Lowering the airway surface liquid (ASL) salt concentration has been shown to increase activity of salt sensitive antimicrobial peptides.

Xylitol is a 5-carbon sugar that can lower the ASL salt concentration, thus enhancing innate immunity. In this study, the investigators propose to test the safety and tolerability of aerosolized xylitol used daily for 2 weeks in subjects with cystic fibrosis. In a pilot, 2-week study, 60 subjects with cystic fibrosis with an FEV1(Forced expiratory volume in 1 second ) >30% predicted will be randomized to receive aerosolized 7% hypertonic saline (5 ml) or 15% xylitol, (5 ml) twice a day for 14 days. The primary outcomes will be safety as assessed by FEV1 change from baseline, adverse events and respiratory symptom score. Outcomes for trend in efficacy include density of colonization of sputum, time to next exacerbation, sputum cytokines and revised CF quality of life questionnaire.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Joseph Zabner; University of Iowa

Criteria

Inclusion Criteria:

• Subjects with CF (medical record evidence of CFTR(Cystic fibrosis transmembrane conductance regulator) mutation or sweat chloride test or nasal voltage difference, and 1 or more clinical findings of CF),

• Age 12 or greater

• FEV1 > 30% predicted(within the last 14 days and oxygen saturation > 90% on FiO2(fraction of inspired oxygen) ≤ 50%,

• Admitted for an exacerbation,

• Use of effective contraception in women,

• Able to provide written informed consent.

Exclusion Criteria:

• Pregnancy,

• History of asthma based on methacholine challenge or bronchial hyperresponsiveness on PFTS(Pulmonary Function Test),

• Hemoptysis more than 60 mL within the last 30 days,

• Use of any investigational study drug within the last 30 days,

• Initiation of hypertonic saline within the last 30 days,

• A serum creatinine 2 mg/dl or more

• Active malignancy in the last year

• Antibiotics for CF exacerbation as an outpatient in the last 2 weeks

• B cepacia colonization

• Waiting list for lung transplant

• Lack of FEV1 data from the last 14 days

• Previous participation in this study