Infant Study of Inhaled Saline in Cystic Fibrosis

• Status: Completed
• Trial end date: 2011-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess whether 7% hypertonic saline (HS) is an effective and safe therapy in infants and young children with CF.

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

Details

• Lead Sponsor: CF Therapeutics Development Network Coordinating Center
• Collaborators: Cystic Fibrosis Foundation; Cystic Fibrosis Foundation Therapeutics; National Heart, Lung, and Blood Institute (NHLBI)

Criteria

Inclusion Criteria:

• Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND either: (a) A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis or (b) A genotype with two identifiable CF-causing mutations

• Informed consent by parent or legal guardian

• Age 4 months to < 60 months at Enrollment visit. If participating in Infant Pulmonary Function testing (selected sites), age 4 months to < 16 months at Enrollment visit.

• Ability to comply with medication use, study visits, and study procedures as judged by the site investigator

Exclusion Criteria:

• Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 1 week preceding Enrollment visit

• Acute wheezing at Enrollment visit (prior to HS test dose), or at Infant PFT visit (prior to infant pulmonary function testing), as applicable

• Oxygen saturation < 95% (< 90% in centers located above 4000 feet elevation) at Enrollment visit (prior to HS test dose) or at Infant PFT visit (prior to infant pulmonary function testing), as applicable

• Other major organ dysfunction, excluding pancreatic dysfunction

• Physical findings that would compromise the safety of the subject or the quality of the study data as determined by the site investigator

• Investigational drug use within 30 days prior to Enrollment visit, or within 30 days prior to Infant PFT visit as applicable

• Treatment with inhaled hypertonic saline at any concentration within 30 days of Enrollment visit, or within 30 days prior to Infant PFT visit as applicable

• Chronic lung disease not related to CF

• Intolerance of test dose of HS at Enrollment visit

• A sibling that has been randomized and is still enrolled in ISIS002

Additional Exclusion Criteria for Participation in Infant Pulmonary Function Testing:

• History of adverse reaction to sedation

• Clinically significant upper airway obstruction as determined by the Site Investigator (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)

• Severe gastroesophageal reflux, defined as persistent frequent emesis despite anti-reflux therapy

• Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 2 weeks preceding visit