Cystic fibrosis MedDRA version: 19.1 Level: PT Classification code 10011763 Term: Cystic fibrosis lung System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Hypertonic saline
Pharmaceutical Form: Inhalation solution
INN or Proposed INN: sodium chloride 7%
CAS Number: 7647-14-5
Current Sponsor code: sodium chloride 7%
Other descriptive name:
Sponsors
Erasmus MC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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: a) A documented sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT) b) 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 Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 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 Chest CT within 8 months prior to screening or enrolment 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 enrolment visit 10. Chronic lung disease not related to CF 11. Inability to tolerate first dose of study treatment at the enrolment visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Compare the PRAGMA-CF total percent disease (%Dis) score (the volume proportion of the lung with structural airways disease) measured from standardized chest CT at 48 weeks between treatment arms;Secondary Objective: Compare the PRAGMA-CF sub-scores %Bx (the volume proportion of the lung with bronchiectasis) and %TA (the volume proportion of the lung with trapped air), as well as airway-to-artery ratios (AA ratios) measured from chest CT images at 48 weeks between treatment arms 3. Compare the change in LCI, measured by N2 MBW, from baseline to 48 weeks between treatment arms. 4. Elucidate the cross-sectional and longitudinal relationships between measures of structural lung disease evaluated by chest CT (PRAGMA-CF (%Dis, %Bx, %TA) and AA method (AA ratio and airway dimensions), LCI measured by multiple breath washout and clinical outcomes (pulmonary exacerbations, health-related quality of life) over the 48-week treatment period.;Primary end point(s): The difference in PRAGMA-CF %Dis between HS and IS study arm at end of study (48 weeks), adjusted for baseline, measured from standardized chest CT.;Timepoint(s) of evaluation of this end point: During the whole study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): i)The difference in PRAGMA-CF sub-scores, %Dx and %TA, between the baseline CT and the 48 week CT. ii) The absolute number of airways, airway dimensions and AA ratios from TLC CTs, acquired at the 48-week visit. 2. The difference in LCI, measured by N2 MBW, from baseline to 48 weeks 3. Cross-sectional and longitudinal relationships between primary and secondary PRAGMA-CF outcomes (%Dis, %Bx and %TA) and MBW outcomes (LCI), airway dimensions and PRAGMA-CF and MBW outcomes, as well as CFQ-R scores and PRAGMA-CF and MBW outcomes.;Timepoint(s) of evaluation of this end point: During the whole study. | — |
Countries
Australia, Belgium, Denmark, France, Italy, Netherlands, Spain, United States
Outcome results
None listed