Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, Hypertonic Saline, Infants
Brief summary
The objective of the study is to determine whether or not inhalation of hypertonic saline will be tolerated by infants with cystic fibrosis and the effect of inhalation on their lung function.
Detailed description
Cystic fibrosis lung disease is characterized by mucous retention which favors secondary bacterial infection and inflammation, which leads to lung damage and ultimately respiratory failure. Classically, therapeutic interventions are aimed to improve mucociliary clearance, to reduce both bacterial load and lower airway inflammation. Hypertonic saline (HS) has been used for the induction of sputum production in all age groups to obtain secretions from the lower respiratory for diagnostic purposes. Hypertonic saline is also used in older children with CF, who do not produce sputum spontaneously, to obtain representative samples for microbiology. There is evidence from studies in patients with cystic fibrosis that HS can improve mucociliary clearance. The improvement was more impressive in areas that were well ventilated, making it likely that HS will work better in patients with relatively preserved pulmonary function. Newer evidence also suggests that the osmotic effect on the airway surface that was expected to be short lived, may actually persist for longer time periods (up to 8 hours). All these data indicate that HS may be a useful agent in the treatment of CF patients. As the effect on mucociliary clearance was found to be better in areas with adequate ventilation, it is logical to assume that treatment with HS may be most efficacious when initiated early in the disease process. So far, no data on the tolerability of inhalation of HS are available for infants with cystic fibrosis. However, evidence from infants with AIDS as well as recent studies in infants with bronchiolitis suggest that hypertonic saline can be safely administered by inhalation in infants. Nevertheless, proof of tolerability in CF infants is a prerequisite for longer term studies of HS in this age group. In older children, tolerability has been tested by measuring pulmonary function both before and after inhalation of HS saline. Similar data are not yet available for infants.
Interventions
DRUGHypertonic Saline
5 ml of 7% saline will be administered via mask with Pari LC Plus nebuliser and a Pari Ultra Ned compressor.
Sponsors
The Hospital for Sick Children
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Months to 2 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of cystic fibrosis * Age 2 months to 2 years * Routinely scheduled infant pulmonary function test. * Clinical stability (no respirator tract infection for 4 weeks prior to study
Exclusion criteria
* Acute respiratory symptoms * Wheezing at the time of study * Supplemental oxygen therapy * Oxygen saturation \< 95 % on room air
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Decrease in FEV0.5 After Inhalation of Hypertonic Saline | 100 minutes | Number of participants with a decrease in FEV0.5 by 0% or more after inhalation of hypertonic saline. The number of participants with a change in FEV0.5 of \*more than\* 0% is reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Oxygen Saturation Below 92% | 10 minutes | Reduction of oxygen saturation below 92 % 10 minutes after inhalation of hypertonic saline. This change would be considered to reflect an abnormally low oxygen concentration and could reflect a negative impact of inhalation |
Other
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Newly Detected Bacterial Pathogens on Throat Swab After Hypertonic Saline Inhalation | 10 minutes after completion of inhalation | Diagnostic yield of a throat swab for microbiological diagnosis of bacteria as measured by the number of participants with newly detected bacterial pathogens on respiratory culture after inhalation of hypertonic saline compared to previously performed throat swabs at the last clinic visit |
| Number of Participants With Wheezing After Inhalation of Hypertonic Saline | 15 minutes | audible wheezing on auscultation 15 minutes after administration |
Countries
Canada
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Single Arm Study Hypertonic Saline: 5 ml of 7% saline will be administered via mask with Pari LC Plus nebuliser and a Pari Ultra Ned compressor. | 13 |
| Total | 13 |
Baseline characteristics
| Characteristic | Single Arm Study |
|---|---|
| Age, Customized Age, continuous | 80.7 weeks STANDARD_DEVIATION 31.4 |
| Region of Enrollment Canada | 13 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 13 |
| other Total, other adverse events | 0 / 13 |
| serious Total, serious adverse events | 0 / 13 |
Outcome results
Primary
Decrease in FEV0.5 After Inhalation of Hypertonic Saline
Number of participants with a decrease in FEV0.5 by 0% or more after inhalation of hypertonic saline. The number of participants with a change in FEV0.5 of \*more than\* 0% is reported.
Time frame: 100 minutes
Population: 2 infants had to excluded, since they woke up before administration of the study medication and thus lung function measurements after administration could not be performed
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Decrease in FEV05 | Decrease in FEV0.5 After Inhalation of Hypertonic Saline | 0 participants |
Secondary
Oxygen Saturation Below 92%
Reduction of oxygen saturation below 92 % 10 minutes after inhalation of hypertonic saline. This change would be considered to reflect an abnormally low oxygen concentration and could reflect a negative impact of inhalation
Time frame: 10 minutes
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Decrease in FEV05 | Oxygen Saturation Below 92% | 0 participants |
Other Pre-specified
Number of Participants With Newly Detected Bacterial Pathogens on Throat Swab After Hypertonic Saline Inhalation
Diagnostic yield of a throat swab for microbiological diagnosis of bacteria as measured by the number of participants with newly detected bacterial pathogens on respiratory culture after inhalation of hypertonic saline compared to previously performed throat swabs at the last clinic visit
Time frame: 10 minutes after completion of inhalation
Population: CF infants
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Decrease in FEV05 | Number of Participants With Newly Detected Bacterial Pathogens on Throat Swab After Hypertonic Saline Inhalation | 0 participants |
Other Pre-specified
Number of Participants With Wheezing After Inhalation of Hypertonic Saline
audible wheezing on auscultation 15 minutes after administration
Time frame: 15 minutes
Population: Individuals with lung function data after inhalation
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Decrease in FEV05 | Number of Participants With Wheezing After Inhalation of Hypertonic Saline | 0 percentage of participants |