Condition category
Genetic Diseases
Date applied
04/05/2016
Date assigned
06/07/2016
Last edited
06/07/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

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

Trial website

Contact information

Type

Scientific

Primary contact

Dr Raffaella Nenna

ORCID ID

Contact details

Department of Pediatrics and Pediatric Neuropsychiatry
Sapienza University of Rome
Viale Regina Elena 324
Rome
00161
Italy

Additional identifiers

EudraCT 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

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

Trial setting

Hospitals

Trial 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

Phase

Not Applicable

Drug names

Hypertonic saline

Primary outcome measures

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 trial start date

01/02/2010

Overall trial end date

01/02/2015

Reason abandoned

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

Patient

Age group

Child

Gender

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

Trial 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

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

Participant level data

To be made available at a later date

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes