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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
150

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Mar 2015

Longer than P75 for not_applicable

Geographic Reach
2 countries

25 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 27, 2015

Completed
2 days until next milestone

Study Start

First participant enrolled

March 1, 2015

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 4, 2015

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2018

Completed
Last Updated

January 14, 2020

Status Verified

December 1, 2019

Enrollment Period

3.5 years

First QC Date

February 27, 2015

Last Update Submit

January 10, 2020

Conditions

Keywords

Cystic FibrosisHypertonic SalineInhaled SalineDigestive System DiseasesGenetic Diseases, InbornLung DiseasesPancreatic DiseasesPathologic ProcessesRespiratory Tract Diseases

Outcome Measures

Primary Outcomes (1)

  • Change in Lung Clearance Index (LCI)

    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.

    48 weeks

Secondary Outcomes (5)

  • Change in Forced Expiratory Volume (FEV)

    48 weeks

  • Pulmonary exacerbation rate

    48 weeks

  • Health-related quality of life

    48 weeks

  • Respiratory Signs

    48 weeks

  • Respiratory Pathogen Rate

    48 weeks

Study Arms (2)

Active Treatment Group

EXPERIMENTAL

7% Hypertonic Saline administered via inhalation twice daily for 48 weeks

Drug: 7% Hypertonic Saline (HS)

Control Group

ACTIVE COMPARATOR

0.9% Isotonic Saline administered via inhalation twice daily for 48 weeks

Drug: 0.9% Isotonic Saline (IS)

Interventions

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.

Also known as: Hyper-Sal™, inhaled saline
Active Treatment Group

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.

Also known as: Normal saline
Control Group

Eligibility Criteria

Age3 Years - 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

University of Alabama-Birmingham: Pulmonary, Allergy and Critical Care Medicine

Birmingham, Alabama, 35233, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

Nemours Children's Hospital

Orlando, Florida, 32827, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children-Indiana U Med Center

Indianapolis, Indiana, 46202, United States

Location

University of Iowa, Department of Pediatrics

Iowa City, Iowa, 52242, United States

Location

Johns Hopkins University: Pediatric Pulmonary

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Women & Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599-7217, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Rainbow Babies Children's Hospital and Case Western Reserve University School of Medicine

Cleveland, Ohio, 44212, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia, UPenn

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Texas Children's Hospital and Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

Hospital for Sick Kids

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (3)

  • Rosenfeld M, Ratjen F, Brumback L, Daniel S, Rowbotham R, McNamara S, Johnson R, Kronmal R, Davis SD; ISIS Study Group. Inhaled hypertonic saline in infants and children younger than 6 years with cystic fibrosis: the ISIS randomized controlled trial. JAMA. 2012 Jun 6;307(21):2269-77. doi: 10.1001/jama.2012.5214.

    PMID: 22610452BACKGROUND
  • Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.

    PMID: 20435858BACKGROUND
  • Ratjen F, Davis SD, Stanojevic S, Kronmal RA, Hinckley Stukovsky KD, Jorgensen N, Rosenfeld M; SHIP Study Group. Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Respir Med. 2019 Sep;7(9):802-809. doi: 10.1016/S2213-2600(19)30187-0. Epub 2019 Jun 6.

MeSH Terms

Conditions

Cystic FibrosisDigestive System DiseasesGenetic Diseases, InbornLung DiseasesPancreatic DiseasesPathologic ProcessesRespiratory Tract Diseases

Interventions

Saline Solution, HypertonicSodium ChlorideSaline Solution

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Hypertonic SolutionsSolutionsPharmaceutical PreparationsChloridesHydrochloric AcidChlorine CompoundsInorganic ChemicalsSodium CompoundsCrystalloid SolutionsIsotonic Solutions

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 27, 2015

First Posted

March 4, 2015

Study Start

March 1, 2015

Primary Completion

August 31, 2018

Study Completion

August 31, 2018

Last Updated

January 14, 2020

Record last verified: 2019-12

Locations