Saline Hypertonic in Preschoolers + CT

• Status: Completed
• Trial end date: 2021-06-25
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6) with cystic fibrosis.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center
• Collaborators: Cystic Fibrosis Foundation; Cystic Fibrosis Foundation Therapeutics

Criteria

Inclusion Criteria:

1. Diagnosis of CF as evidenced by one or more clinical features consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:

1. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)

2. A documented genotype with two disease-causing mutations in the CFTR gene

2. Informed consent by parent or legal guardian

3. Age ≥ 36 months and ≤72 months at screening visit

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

5. Ability to cooperate with chest CT at the enrollment visit as determined by the lung function technician

Exclusion Criteria:

1. Chest CT within 8 months prior to the Screening visit

2. Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset within 3 weeks preceding screening or enrollment visit

3. Acute wheezing at screening or enrollment visit

4. Oxygen saturation < 95% (<90% in centers located above 4000 feet elevation) at screening or enrollment visit

5. Other major organ dysfunction, excluding pancreatic dysfunction

6. Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator

7. Investigational drug use within 30 days prior to screening or enrollment visit

8. Treatment with inhaled HS at any concentration within 30 days prior to screening or enrollment visit

9. Initiation (i.e. new prescription) of any inhaled hydrating agent such as mannitol or mucolytic agents such as dornase alpha within 30 days prior to the screening or enrollment visit

10. Chronic lung disease not related to CF

11. Inability to tolerate first dose of study treatment at the enrollment visit