Saline Hypertonic in Preschoolers with Cystic Fibrosis and lung structure as measured by computed tomography (CT). SHIP-CT study.
A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks on lung structure in patients with Cystic Fibrosis at 3-6 years of age, in parallel with the North American SHIP clinical trial, as measured by computed tomography (CT).
Telethon Kids Institute
120 participants
Nov 9, 2017
Interventional
Conditions
Summary
People with cystic fibrosis (CF) often have thick mucus in the airways of the lungs that is hard to cough up. The mucus builds up and eventually leads to chronic cough and lung infections. Research has shown that even young children with CF may have thickened mucus in the lungs. Inhaling a concentrated salt solution, called hypertonic saline (HS), may help thin the mucus in the lungs. Thinning the mucus can make it easier to cough up. This helps to clear the lungs and improve lung health. Research studies about the safety and effectiveness of inhaled HS have been done in adults and children with CF 6 years of age and older. Patients in these studies took HS for up to a year. HS appears to be a safe treatment in these age groups. The main side effects were cough, throat irritation, and wheezing. The use of HS in older children and adults decreased the need for antibiotics for acute respiratory infections. It also improved lung function and quality of life. HS is now routinely used by many CF patients over 6 years of age. Because HS treats a very early step in the chain of events that leads to chronic lung problems in people with CF, it may be particularly helpful when started early in life. Based on several studies, HS appears to be safe in children less than 6 years of age, but its effectiveness has been difficult to measure. In a previous study, children less than 6 year-old receiving HS had the same number of lung infections as children receiving normal saline. However, we think that children this young need a more sensitive test to see if HS works in preventing lung damage. In this study Computed Tomography (CT) scans will be used to get an image of the lung. In addition a new type of breathing test, called Multiple Breath Washout (MBW), will be used. This is relatively easy for preschool children to perform, and may allow us to measure the effectiveness of HS in preschool children with CF. The MBW tests measures lung function. This study will be conducted in parallel with the North American SHIP001 clinical study, registered on Clinicaltrials.gov NCT02378467.
Eligibility
Inclusion Criteria7
- 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 greater than or equal to 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 greater than or equal to 36 months and less than or equal to 72 months at screening visit
- Ability to comply with medication use, study visits and study procedures as judged by the site investigator
- Ability to perform a chest CT at the enrolment visit
Exclusion Criteria11
- 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 enrolment visit
- Oxygen saturation < 95% (<90% in centres located above 4000 feet elevation) at screening or enrolment 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 HS 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 Enrolment visit
- Chronic lung disease not related to CF
- Inability to tolerate first dose of study treatment at the enrolment visit
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Interventions
Experimental: Active Treatment Group 7% Hypertonic Saline administered via inhalation twice daily for 48 weeks. Drug - 7% Hypertonic Saline (HS) Administered via inhalation of 4mL twice daily for 48 weeks. The delivery system is a PARI Sprint Junior nebulizer with a PARI Baby face mask or mouthpiece driven by a PARI compressor.
Locations(13)
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ACTRN12615001067561