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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02950883




Registration number
NCT02950883
Ethics application status
Date submitted
27/10/2016
Date registered
1/11/2016
Date last updated
14/01/2020

Titles & IDs
Public title
Saline Hypertonic in Preschoolers + CT
Scientific title
Saline Hypertonic in Preschoolers With Cystic Fibrosis and Lung Structure as Measured by Computed Tomography (CT)
Secondary ID [1] 0 0
SHIP002
Universal Trial Number (UTN)
Trial acronym
SHIP-CT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Active Treatment Group 7% Hypertonic Saline
Treatment: Drugs - Control Group 0.9% Isotonic Saline

Experimental: Active Treatment Group - 7% Hypertonic Saline administered via inhalation twice daily for 48 weeks

Active Comparator: Control Group - 0.9% Isotonic Saline administered via inhalation twice daily for 48 weeks


Treatment: Drugs: Active Treatment Group 7% Hypertonic Saline
Drug: 7% Hypertonic Saline (HS) 4 mL of HS will be administered via inhalation 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 (PARI Vios® Pro in USA, PARI BOY SX in Australia and Europe).
Other Names:
Hyper-Salâ„¢, inhaled saline

Treatment: Drugs: Control Group 0.9% Isotonic Saline
Drug: 0.9% Isotonic Saline (IS) 4 mL of IS will be administered via inhalation twice daily for 48 weeks The delivery system is the same as that for the test product.
Other Names: Normal saline

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Chest CT - The difference in PRAGMA-CF %Dis between HS and IS study arm at end of study (48 weeks), adjusted for baseline, measured from standardized chest CT.
Timepoint [1] 0 0
48 weeks
Secondary outcome [1] 0 0
PRAGMA-CF Sub-scores - i) The difference in PRAGMA-CF sub-scores, %Bx (the volume proportion of the lung with bronchiectasis) and %TA (the volume proportion of the lung with trapped air), between the baseline CT and the 48 week CT.
ii) The absolute number of airways, airway dimensions and AA ratios from TLC CTs, acquired at the 48-week visit.
Timepoint [1] 0 0
48 weeks
Secondary outcome [2] 0 0
Lung Clearance Index (LCI) - The difference in LCI, measured by N2 MBW, from baseline to 48 weeks
Timepoint [2] 0 0
48 weeks
Secondary outcome [3] 0 0
Cross-sectional and longitudinal relationships - Cross-sectional and longitudinal relationships between primary and secondary PRAGMA-CF outcomes (%Dis, %Bx and %TA) and MBW outcomes (LCI), airway dimensions and PRAGMA-CF and MBW outcomes, as well as CFQ-R scores and PRAGMA-CF and MBW
Timepoint [3] 0 0
48 weeks

Eligibility
Key inclusion criteria
1. Diagnosis of CF as evidenced by one or more clinical features consistent with the CF
phenotype or positive CF newborn screen AND one or more of the following criteria:

1. A documented sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis
(QPIT)

2. A documented genotype with two disease-causing mutations in the CFTR gene

2. Informed consent by parent or legal guardian

3. Age = 36 months and =72 months at screening visit

4. Ability to comply with medication use, study visits and study procedures as judged by
the site investigator

5. Ability to cooperate with chest CT at the enrollment visit as determined by the lung
function technician
Minimum age
3 Years
Maximum age
5 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Chest CT within 8 months prior to the Screening visit

2. Acute intercurrent respiratory infection, defined as an increase in cough, wheezing,
or respiratory rate with onset within 3 weeks preceding screening or enrollment visit

3. Acute wheezing at screening or enrollment visit

4. Oxygen saturation < 95% (<90% in centers located above 4000 feet elevation) at
screening or enrollment visit

5. Other major organ dysfunction, excluding pancreatic dysfunction

6. Physical findings that would compromise the safety of the participant or the quality
of the study data as determined by site investigator

7. Investigational drug use within 30 days prior to screening or enrollment visit

8. Treatment with inhaled HS at any concentration within 30 days prior to screening or
enrollment visit

9. Initiation (i.e. new prescription) of any inhaled hydrating agent such as mannitol or
mucolytic agents such as dornase alpha within 30 days prior to the screening or
enrollment visit

10. Chronic lung disease not related to CF

11. Inability to tolerate first dose of study treatment at the enrollment visit

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Women's and Children Hospital - Adelaide
Recruitment hospital [2] 0 0
Lady Cilento Children's Hospital - Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [4] 0 0
John Hunter Children's Hospital - Newcastle
Recruitment hospital [5] 0 0
Children's Hospital at Westmead - Sydney
Recruitment hospital [6] 0 0
Sydney Children's Hospital at Randwick - Sydney
Recruitment hospital [7] 0 0
Perth Children's Hospital - West Perth
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Newcastle
Recruitment postcode(s) [5] 0 0
- Sydney
Recruitment postcode(s) [6] 0 0
- West Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
North Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Oregon
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Washington
Country [8] 0 0
Belgium
State/province [8] 0 0
Brussels
Country [9] 0 0
Belgium
State/province [9] 0 0
Leuven
Country [10] 0 0
Canada
State/province [10] 0 0
British Columbia
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Denmark
State/province [12] 0 0
Copenhagen
Country [13] 0 0
France
State/province [13] 0 0
Lyon
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Italy
State/province [15] 0 0
Roma
Country [16] 0 0
Italy
State/province [16] 0 0
Verona
Country [17] 0 0
Netherlands
State/province [17] 0 0
Rotterdam
Country [18] 0 0
Spain
State/province [18] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Other
Name
University of Washington, the Collaborative Health Studies Coordinating Center
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Cystic Fibrosis Foundation
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice
daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in
comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6)
with cystic fibrosis.
Trial website
https://clinicaltrials.gov/show/NCT02950883
Trial related presentations / publications
Rosenow T, Oudraad MC, Murray CP, Turkovic L, Kuo W, de Bruijne M, Ranganathan SC, Tiddens HA, Stick SM; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF). PRAGMA-CF. A Quantitative Structural Lung Disease Computed Tomography Outcome in Young Children with Cystic Fibrosis. Am J Respir Crit Care Med. 2015 May 15;191(10):1158-65. doi: 10.1164/rccm.201501-0061OC.
Ramsey KA, Rosenow T, Turkovic L, Skoric B, Banton G, Adams AM, Simpson SJ, Murray C, Ranganathan SC, Stick SM, Hall GL; AREST CF. Lung Clearance Index and Structural Lung Disease on Computed Tomography in Early Cystic Fibrosis. Am J Respir Crit Care Med. 2016 Jan 1;193(1):60-7. doi: 10.1164/rccm.201507-1409OC.
Public notes

Contacts
Principal investigator
Name 0 0
Harm Tiddens, MD, PhD
Address 0 0
Erasmus Medical Centre, Rotterdam
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications