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Saline Hypertonic in Preschoolers (SHIP)

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ClinicalTrials.gov Identifier: NCT02378467
Recruitment Status : Active, not recruiting
First Posted : March 4, 2015
Last Update Posted : December 4, 2017
Sponsor:
Collaborator:
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
University of Washington, the Collaborative Health Studies Coordinating Center

Brief Summary:
The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks improves the lung clearance index by multiple breath nitrogen washout in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 5) with cystic fibrosis.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: 7% Hypertonic Saline (HS) Drug: 0.9% Isotonic Saline (IS) Not Applicable

Detailed Description:

A growing body of evidence supports the importance of intervention in cystic fibrosis (CF) lung disease during early childhood, in order to potentially delay or prevent irreversible lung damage. Yet, aside from antimicrobial therapies, the CF community has no clinical trial evidence base with which to guide chronic pulmonary therapies in preschool children. Hypertonic saline (HS) is the most attractive chronic maintenance therapy to investigate in preschool children because it addresses defective mucociliary clearance, an early step in the cascade of events leading to CF lung disease that is expected to be abnormal prior to the onset of airway infection and inflammation.

Based on several studies, HS appears to be safe in children less than 6 years of age, but its effectiveness has been difficult to measure. In a previous study (ISIS), children less than 6 years old receiving HS had the same number of lung infections as children receiving a control treatment. However, the investigators think that children this young need a more sensitive test, such as lung function testing, to see if HS works in preventing lung damage. Multiple Breath Washout (MBW) is a relatively easy lung function test to perform with preschool children. It calculates a measurement called the Lung Clearance Index (LCI), a sensitive measure of airway inhomogeneity. In a sub-study of the ISIS study in 25 children, LCI improved in children that inhaled HS twice daily for 48 weeks.

This is a multicenter, randomized, double-blind, controlled, parallel group trial assessing LCI in children with CF ages 3 to 5 at enrollment. Participants will be randomized 1:1 to receive 7% HS (treatment arm) vs. 0.9 % isotonic saline (control arm) administered twice daily via jet nebulizer for 48 weeks. Study visits will occur at Screening, Enrollment, and at Weeks 12, 24, 36 and 48. Contact with a parent or legal guardian to assess adherence and interim medical history will occur at 1, 4 and 8 weeks after enrollment and then quarterly between subsequent study visits. Except for the screening visit, study visits will occur on a quarterly basis.

Total duration of participation will be up to 52 weeks. As enrollment will occur over approximately 18 months, total duration of the study is expected to be up to 30 months (18 months enrollment plus 12 months for the last participants to complete study participation).


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 150 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Saline Hypertonic in Preschoolers
Study Start Date : March 2015
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : February 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Active Treatment Group
7% Hypertonic Saline administered via inhalation twice daily for 48 weeks
Drug: 7% Hypertonic Saline (HS)
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 Vios® compressor.
Other Name: Hyper-Sal™, inhaled saline

Active Comparator: Control Group
0.9% Isotonic Saline administered via inhalation twice daily for 48 weeks
Drug: 0.9% Isotonic Saline (IS)
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 Vios® compressor.
Other Name: Normal saline




Primary Outcome Measures :
  1. Change in Lung Clearance Index (LCI) [ Time Frame: 48 weeks ]
    Change in Lung Clearance Index (LCI) from baseline to 48 weeks measured by N2 Multiple Breath Washout (MBW) between subjects randomized to HS and IS.


Secondary Outcome Measures :
  1. Change in Forced Expiratory Volume (FEV) [ Time Frame: 48 weeks ]
    Change in FEV 0.75 measured by preschool spirometry between subjects randomized to HS and IS.

  2. Pulmonary exacerbation rate [ Time Frame: 48 weeks ]
    Protocol defined pulmonary exacerbation rate.

  3. Health-related quality of life [ Time Frame: 48 weeks ]
    Health-related quality of life as measured by the modified parent-reported Cystic Fibrosis Questionnaire-Revised (CFQ-R) for preschoolers.

  4. Respiratory Signs [ Time Frame: 48 weeks ]
    Parent observation of respiratory signs as measured by the Cystic Fibrosis Respiratory Sign Diary for ages 0-6 (CFRSD0-6).

  5. Respiratory Pathogen Rate [ Time Frame: 48 weeks ]
    Rates of treatment emergent CF respiratory pathogens from clinical respiratory cultures.



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:
  • A documented sweat chloride ≥ 60 milliequivalents of solute per litre (mEq/L) by quantitative pilocarpine iontophoresis (QPIT)
  • A documented genotype with two disease-causing mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene
  • Informed consent by parent or legal guardian
  • Age ≥ 36 months and ≤72 months at Screening visit
  • Ability to comply with medication use, study visits and study procedures as judged by the site investigator
  • Ability to perform technically acceptable MBW measurements at the screening and enrollment visits

Exclusion Criteria:

  • Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset within 3 weeks preceding Screening or Enrollment visit
  • Acute wheezing at Screening or Enrollment visit
  • Oxygen saturation < 95% (<90% in centers located above 4000 feet elevation) at Screening or Enrollment visit
  • Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator
  • Investigational drug use within 30 days prior to Screening or Enrollment visit
  • Treatment with inhaled hypertonic saline at any concentration within 30 days prior to Screening or Enrollment visit
  • Chronic lung disease not related to CF
  • Inability to tolerate first dose of study treatment at the Enrollment visit

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02378467


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Sponsors and Collaborators
University of Washington, the Collaborative Health Studies Coordinating Center
Cystic Fibrosis Foundation Therapeutics
Investigators
Principal Investigator: Stephanie Davis, MD Indiana University
Principal Investigator: Richard A Kronmal, PhD University of Washington
Principal Investigator: Felix Ratjen, MD, PhD, FRCPC Hospital for Sick Kids, Toronto
Principal Investigator: Margaret Rosenfeld, MD, MPH Seattle Children's Hospital

Publications:
Responsible Party: University of Washington, the Collaborative Health Studies Coordinating Center
ClinicalTrials.gov Identifier: NCT02378467     History of Changes
Other Study ID Numbers: SHIP001
First Posted: March 4, 2015    Key Record Dates
Last Update Posted: December 4, 2017
Last Verified: November 2017

Keywords provided by University of Washington, the Collaborative Health Studies Coordinating Center:
Cystic Fibrosis
Hypertonic Saline
Inhaled Saline
Digestive System Diseases
Genetic Diseases, Inborn
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases