A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with Cystic Fibrosis at 3-6 years of age in parallel with the North American SHIP clinical trial
- Conditions
- CF10027664
- Registration Number
- NL-OMON46189
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 14
Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria: a) A documented sweat chloride * 60 mEq/L by quantitative pilocarpineiontophoresis (QPIT) b) A documented genotype with two disease-causing mutations in the CFTR gene ;Informed consent by parent or legal guardian;Age * 36 months and *72 months at Screening visit ;Ability to comply with medication use, study visits and study procedures as judged by the site investigator;***Ability to execute a technician controlled or spirometer controlled chest CT scan***????
Chest CT within 8 months prior to the Screening visit;Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset within 3 weeks preceding Screening or Enrolment visit;Acute wheezing at Screening or Enrollment visit ;Oxygen saturation < 95% (<90% at centres above 4000 feet elevation) at Screening or Enrollment visit ;Other major organ dysfunction, excluding pancreatic dysfunction;Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator;Investigational drug use within 30 days prior to Screening or Enrolment visit;Treatment with inhaled hypertonic saline at any concentration within 30 days prior to Screening or Enrolment visit;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 Enrollment visit;Chronic lung disease not related to CF ;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 <p>The difference in PRAGMA-CF %Dis between HS and IS study arm at end of study<br /><br>(48 weeks), measured from standardized chest CT.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Longitudinal change in airway disease (%Dis), bronchiectasis (%Bx) trapped air<br /><br>(%TA), and airway dimensions, as well as the proportion of patients with<br /><br>bronchiectasis progression established by %BX and AA-system, airway wall<br /><br>thickness established by the AA-system, from baseline to end of study as<br /><br>established by PRAGMA-CF and the AA-system, on chest CTs*<br /><br>Longitudinal change in LCI from baseline to 48 weeks measured by N2 MBW<br /><br>Protocol defined pulmonary exacerbation rate<br /><br>Health-related quality of life as measured by the modified parent-reported<br /><br>CFQ-R for preschoolers (excluding European sites)</p><br>