Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Registration number
ACTRN12616000363482
Ethics application status
Approved
Date submitted
15/03/2016
Date registered
21/03/2016
Date last updated
14/05/2019
Date data sharing statement initially provided
14/05/2019
Date results information initially provided
14/05/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Humidified high flow air for children and young people with Cystic Fibrosis (CF): A pilot study
Scientific title
Pilot Study of Humidified High Flow Air via Nasal Cannulae (HHF) During Sleep for Children and Young People with Cystic Fibrosis (CF)
Secondary ID [1] 288762 0
nil known
Universal Trial Number (UTN)
U1111-1174-6606
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 298010 0
Condition category
Condition code
Respiratory 298170 298170 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The primary intervention for this study is humidified high flow (HHF) air via nasal canulae during sleep. The air will be delivered at 100% relative humidity. No additional oxygen will be used. This is a crossover study and participants will receive in randomised order i) five nights of usual care and ii) five nights of HHF therapy in addition to usual care. HHF will be delivered at an individualized rate of between 20 and 45 L/min depending on participant weight. The study will be conducted in the participant's home. Adherence will not be monitored. Treatment acceptability will be assessed by questionnaire.
Intervention code [1] 294201 0
Treatment: Devices
Comparator / control treatment
Comparator is usual treatment (no placebo). This will be determined by the usual treating phsyician and consists of twice daily chest physiotherapy with oral vitamin and calorific supplemenation. Participants may be on oral azithromycin. Some participants will continue on their usual nebulised pulmozyme, hypertonic saline or nebulised antibiotics.
Control group
Active

Outcomes
Primary outcome [1] 297680 0
Respiratory rate in sleep measured during limited channel polysomnography in the home.
Timepoint [1] 297680 0
Overnight during sleep on the 5th night of each treatment period.
Secondary outcome [1] 321809 0
Oxygen saturation in sleep measured during limited channel polysomnography in the home.
Timepoint [1] 321809 0
Overnight during sleep on the 5th night of each treatment period.
Secondary outcome [2] 321810 0
Heart rate in sleep measured during limited channel polysomnography in the home.
Timepoint [2] 321810 0
Overnight during sleep on the 5th night of each treatment period.
Secondary outcome [3] 321811 0
Daytime sleepiness (ESS questionnaire)
Timepoint [3] 321811 0
In the morning after the 5th night of therapy of each treatment period
Secondary outcome [4] 321812 0
Treatment satisfaction (unvalidated questionnaire )
Timepoint [4] 321812 0
In the morning after the 5th night of therapy of each treatment period
Secondary outcome [5] 321970 0
Sleep quality (Leeds Sleep Evaluation Questionnaire)
Timepoint [5] 321970 0
Each morning after sleep for 5 mornings during each treatment period
Secondary outcome [6] 321971 0
Breathlessness and cough (both from Cystic Fibrosis Breathlessness Assessment Visual Analog Scale) - composite secondary outcome
Timepoint [6] 321971 0
Each morning after sleep for 5 mornings during each treatment period
Secondary outcome [7] 321972 0
Apnoea and hypopnoea scored during limited channel home polysomnography - composite secondary outcome
Timepoint [7] 321972 0
Overnight during sleep on 5th night of each treatment period

Eligibility
Key inclusion criteria
- Children and young people aged 6 years to 25 years with a confirmed diagnosis of cystic fibrosis
- Mild or moderate disease
- The participant is in a stable period of well-being.
Minimum age
6 Years
Maximum age
25 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- FEV1 <= 30% predicted for height, age and gender
- Participant already requires respiratory support in sleep with oxygen, HHF, CPAP or VPAP.
- Participant under the care of the Lung Transplant team
- Exacerbation or symptoms of an exacerbation within the last 4 weeks
- Participant has additional complex diagnosis such as immune deficiency, cardiac disease or malignancy

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Sealed envelope
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Computer generated
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Crossover
Other design features
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
Paired t-test with repeated measures analysis for outcomes measured on a daily basis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
4/04/2016
Actual
1/12/2016
Date of last participant enrolment
Anticipated
19/11/2018
Actual
30/07/2018
Date of last data collection
Anticipated
24/12/2018
Actual
17/08/2018
Sample size
Target
15
Accrual to date
Final
16
Recruitment outside Australia
Country [1] 7707 0
New Zealand
State/province [1] 7707 0

Funding & Sponsors
Funding source category [1] 293125 0
Charities/Societies/Foundations
Name [1] 293125 0
A+ Trust
Country [1] 293125 0
New Zealand
Primary sponsor type
Hospital
Name
Auckland District Health Board (contact Dr David McNamara)
Address
Private Bag 92024
Auckland 1023
Country
New Zealand
Secondary sponsor category [1] 291919 0
None
Name [1] 291919 0
None
Address [1] 291919 0
None
Country [1] 291919 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 294620 0
Northern B Health and Disability Ethics Committee
Ethics committee address [1] 294620 0
Ministry of Health
Freyberg Building
20 Aitken Street
PO Box 5013
Wellington
6011
Ethics committee country [1] 294620 0
New Zealand
Date submitted for ethics approval [1] 294620 0
06/01/2016
Approval date [1] 294620 0
04/03/2016
Ethics approval number [1] 294620 0
16/NTB/1

Summary
Brief summary
Cystic fibrosis (CF) is a common genetic disorder affecting approximately 1 in 3000 people in New Zealand. CF results in thickened airway mucus which causes infection, inflammation and loss of lung function with gradual onset of respiratory failure. Individuals with CF have poor growth and failure to thrive occuring through malabsorption of fat and fat-soluble nutrients, but also through increased energy expenditure with increased work of breathing. Unlike healthy controls, persons with CF have an increased respiratory rate and work of breathing during sleep. Reducing this may have long term benefits in improving respiratory health, weight gain and overall quality of life.

Humidified high flow air via nasal prongs (HHF) is a novel but increasingly utilized treatment to decrease work of breathing. It is currently used in the hospital setting, but is a relatively easy treatment to deliver in the home. HHF delivers flows up to 70 L/min via nasal prongs and if delivered through sleep may benefit persons with CF by :
i) Reducing the work of breathing through reduced upper airway resistance and dead space
ii) Improving oxygenation
iii) Increasing hydration of airway secretionsimproving airway secretion clearance
As well as short term benefits – these could result in more substantial long term benefits with improved weight gain and reduced respiratory infections – both directly associated with disease prognosis.

The Starship Respiratory Department is the national centre for children with CF, and the national centre for sleep and home respiratory support providing outreach to other hospitals around New Zealand.
In this - initial randomised controlled cross-over study, we will determine the effect of HHF compared to a usual night of ‘unsupported’ sleep in 15 young persons with CF on respiratory rate, work of breathing, and other physiological parameters as well as measuring sleep quality. We will also determine the acceptability of having this support in the home. If effective and acceptable this will inform the development of a larger multi-centre study measuring the effect of HHF on longerterm outcomes such as weight gain, respiratory infections , ability to exercise, quality of life, and general well-being. HHF is potentially an effective new treatment to be used in the home to prevent disease progression and improve longevity.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 64386 0
Dr David McNamara
Address 64386 0
Starship Children's Hospital
Private Bag 92024
Grafton
Auckland, 1023
Country 64386 0
New Zealand
Phone 64386 0
+6421982483
Fax 64386 0
Email 64386 0
[email protected]
Contact person for public queries
Name 64387 0
Dr David McNamara
Address 64387 0
Starship Children's Hospital
Private Bag 92024
Grafton
Auckland, 1023
Country 64387 0
New Zealand
Phone 64387 0
+6421982483
Fax 64387 0
Email 64387 0
[email protected]
Contact person for scientific queries
Name 64388 0
Dr David McNamara
Address 64388 0
Starship Children's Hospital
Private Bag 92024
Grafton
Auckland, 1023
Country 64388 0
New Zealand
Phone 64388 0
+6421982483
Fax 64388 0
Email 64388 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Privacy requirement of ethical approval


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.