Saline hypertonic in preschoolers with cystic fibrosis and lung structure as measured by computed tomography (CT)

Phase 3

Completed

• Conditions: Cystic fibrosis; Respiratory

• Registration Number: ISRCTN13083896
• Lead Sponsor: Cystic Fibrosis Foundation Therapeutics

Brief Summary

2022 Results article in https://doi.org/10.1016/S2213-2600(21)00546-4 (added 13/05/2022)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-11-05
• Study Completion: 2020-11-01
• Last Update Posted: 23 May 2022

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 120

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:
1.1. A documented sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
1.2. 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 enrolment 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 enrolment visit
3. Acute wheezing at screening or enrolment visit
4. Oxygen saturation < 95% (<90% in centres located above 4000 feet elevation) at screening or enrolment 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 enrolment visit
8. Treatment with inhaled HS at any concentration within 30 days prior to screening or enrolment visit
9. Start of any additional inhaled saline solution at any concentration, or other hydrating agent such as mannitol or mucolytic drug such as dornase alpha within 30 days prior or following the Screening or Enrolment visit
10. Chronic lung disease not related to CF
11. Inability to tolerate first dose of study treatment at the enrolment visit

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The difference in PRAGMA-CF %Dis between HS and IS study arm at end of study (48 weeks), measured from standardized chest CT.

Secondary Outcome Measures

Name	Time	Method
<br> 1. Longitudinal change in airway disease (%Dis), bronchiectasis (%Bx) trapped air (%TA), and airway dimensions, as well as the proportion of patients with bronchiectasis progression established by %BX and AA-system, airway wall thickness established by the AA-system, from baseline to end of study as established by PRAGMA-CF and the AA-system, on chest CTs<br> 2. Longitudinal change in LCI from baseline to 48 weeks measured by N2 MBW<br> 3. Protocol defined pulmonary exacerbation rate<br> 4. Health-related quality of life as measured by the modified parent-reported CFQ-R for preschoolers (excluding European sites)<br>