Plain English Summary
Background and study aims
Cystic fibrosis (CF) is an inherited condition which causes the lungs and digestive system to become blocked with mucus. It is caused by a faulty gene, which is responsible for controlling the movement of water and salts in and out of cells. This leads to a buildup of sticky mucus which clogs the lungs and airways causing breathing difficulties and lung infections, and the digestive system which affects the way food travels through and the ability to absorb nutrients from it. Most people with CF experience problems with lung function and usually are treated with a combination of physiotherapy and medications to prevent lung infections and the buildup of mucus that causes damage. Bronchodilator medications are commonly used in the treatment of CF, as they make breathing easier by relaxing the muscles in the lungs and widening the airways. Following bronchodilation, patients are often given normal saline (salt water) through a nebulizer in order to bring up the mucus blocking their lungs. The aim of this study is to investigate the effectiveness and safety of using hypertonic saline (a solution which is more concentrated than in the body) in children with CF.
Who can participate?
Preschool aged children with cystic fibrosis being treated using bronchodilators and physiotherapy.
What does the study involve?
Participants are randomly allocated to one of two groups who receive two treatments for 16 weeks in a different order. The first treatment involves inhaling a mist of 4ml normal saline (salt water), twice a day for 16 weeks. The second treatment involves inhaling a mist of 4ml hypertonic (more concentrated than blood) saline, twice a day for 16 weeks. At the start of the study and then again after 4, 16, 20 and 32 weeks, participants in both groups complete a number of breathing tests (involving breathing into a machine in various ways) in order to measure how well their lungs are working. Throughout the study, any side effects experienced by the children are recorded on a questionnaire.
What are the possible benefits and risks of participating?
There is a possibility that participants may benefit from improved breathing and lung function. There are no notable risks involved with taking part in this study,
Where is the study run from?
Sapienza University of Rome (Italy)
When is the study starting and how long is it expected to run for?
February 2010 to February 2015
Who is funding the study?
Policlinico Umberto I (Italy)
Who is the main contact?
Dr Rafaella Nenna
Study website
Additional identifiers
EudraCT/CTIS number
IRAS number
ClinicalTrials.gov number
Protocol/serial number
01
Study information
Scientific title
Effects of inhaled hypertonic (7%) saline on lung function test in preschool children with cystic fibrosis: a crossover, randomized clinical trial
Acronym
Study hypothesis
The aim of this study is to evaluate whether inhaled hypertonic saline is effective and safe in children with cystic fibrosis.
Ethics approval(s)
Scientific Ethics Committee, Policlinico Umberto I, 11/03/2010, ref: HS-2009-Prot.1818(11/03/2010)
Study design
Double-blind randomised cross over trial
Primary study design
Interventional
Secondary study design
Randomised cross over trial
Study setting(s)
Hospital
Study type
Treatment
Patient information sheet
No specific participant information sheet available, please use the contact details below to request a further information.
Condition
Cystic fibrosis
Intervention
Participants are randomly allocated to one of two groups who receive two treatments in a random order.
Treatment 1: Participants receive inhalatory administration of 4ml hypertonic saline (HS - 7 % sodium chloride) twice daily for 16 weeks
Treatment 2: Participants receive inhalatory administration of 4ml normal saline (NS - 0.9 % sodium chloride) twice daily for 16 weeks
There is no washout period between the two treatments, and children are followed up after 4, 16, 20 and 32 weeks.
Intervention type
Drug
Pharmaceutical study type(s)
Phase
Not Applicable
Drug/device/biological/vaccine name(s)
Hypertonic saline
Primary outcome measure
1. Airways resistance was measured using interrupter resistance technique at baseline, 4, 16, 20, 32 weeks
2. FVC, FEV1 and FEF25-75 were measured using spirometry at baseline, 4, 16, 20, 32 weeks
Secondary outcome measures
Side effects were registered using a standardized questionnaire created for the purpose of this study throughout the 31 week study period by healthcare providers.
Overall study start date
01/02/2010
Overall study end date
01/02/2015
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Children aged 4-6 years
2. Diagnosis of cystic fibrosis
3. Clinically stable
4. Undergoing a simple therapy based on bronchodilators and physiotherapy
5. No respiratory infections during the treatment or 2 weeks before
Participant type(s)
Patient
Age group
Child
Lower age limit
4 Years
Upper age limit
6 Years
Sex
Both
Target number of participants
12
Participant exclusion criteria
Children with instable medical conditions
Recruitment start date
01/09/2012
Recruitment end date
01/09/2013
Locations
Countries of recruitment
Italy
Study participating centre
Sapienza University of Rome
Cystic Fibrosis Centre
Department of Pediatrics and Infantile Neuropsychiatry
V.le Regina Elena 324
Rome
00161
Italy
Sponsor information
Organisation
Sapienza University of Rome
Sponsor details
Department of Pediatrics and Pediatric Neuropsychiatry
Viale Regina Elena 324
Rome
00161
Italy
Sponsor type
University/education
Website
ROR
Funders
Funder type
University/education
Funder name
Policlinico Umberto I
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Planned publication in the journal BMC Pediatrics
Intention to publish date
01/06/2016
Individual participant data (IPD) sharing plan
IPD sharing plan summary
Data sharing statement to be made available at a later date
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 15/07/2017 | Yes | No |