Funding for this trial has been provided in full or in part by Cystic Fibrosis Foundation Therapeutics, Inc.
Number of Participants Being Recruited:
Single / Multi-Center:
STUDY BACKGROUND INFORMATION:
A growing body of evidence supports the importance of intervention in Cystic Fibrosis (CF) lung disease during infancy, in order to potentially delay or prevent irreversible lung disease. Yet, aside from antimicrobial therapies, the CF community has no clinical trial evidence base with which to guide pulmonary therapies in infants and toddlers. Hypertonic Saline (HS) is the most attractive chronic maintenance therapy to investigate in infants because it addresses defective mucociliary clearance, an early step in the cascade of events leading to CF lung disease that is expected to be abnormal prior to the onset of airway infection and inflammation.
This study is a randomized, parallel group, controlled trial to assess the efficacy and safety of 7% HS inhaled twice daily for 48 weeks among infants with CF 4 to < 16 months of age at enrollment. The primary hypothesis is that, compared to isotonic saline (IS), HS will improve hyperinflation and obstructive lung disease as measured by infant lung function testing. The results of the proposed trial may for the first time provide evidence for early initiation of HS, which, by improving mucociliary clearance, may delay or hinder the cycle of infection and inflammation responsible for progressive airway damage in CF lung disease.
4 Months - 5 Years
P. aeruginosa status:
B. cepacia status:
Other Primary Eligibility Requirements:
Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
FOR MORE INFORMATION:
Sponsor Contact Information:
Rowbotham, Ron (206) 884-7550 firstname.lastname@example.org
Three hundred twenty-one children with CF, aged 4 to 60 months were randomized in the Infant Study of Inhaled Saline in Cystic Fibrosis (ISIS) in the U.S. and Canada. This randomized, double blind trial compared 7% hypertonic saline (active drug) and 0.9% isotonic saline (control agent) inhaled twice daily for 48 weeks. The primary outcome was the rate of pulmonary exacerbations (events per person-year) during the 48-week treatment period. The use of inhaled hypertonic saline compared with isotonic saline did not reduce the rate of pulmonary exacerbations over the course of 48 weeks of treatment.
There was no significant difference in secondary endpoints including height, weight, respiratory rate, oxygen saturation, cough, respiratory symptom scores, or antibiotic use. Infant pulmonary function testing performed in a subgroup (n=73) did not demonstrate significant differences between groups except for the mean change in forced expiratory volume in 0.5 seconds, which was 38 mL greater in the active treatment group.
Adverse event profiles were similar. The most common adverse event of moderate or severe severity was cough (39% of active treatment group, 38% of control group).