Infant Study of Inhaled Saline in Cystic Fibrosis
ISIS
3 other identifiers
interventional
321
2 countries
30
Brief Summary
The purpose of this study is to assess whether 7% hypertonic saline (HS) is an effective and safe therapy in infants and young children with CF.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started Apr 2009
Typical duration for not_applicable
30 active sites
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
July 1, 2008
CompletedFirst Posted
Study publicly available on registry
July 3, 2008
CompletedStudy Start
First participant enrolled
April 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2011
CompletedFebruary 12, 2013
February 1, 2013
2.5 years
July 1, 2008
February 11, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The rate of protocol-defined pulmonary exacerbations requiring treatment with oral, inhaled or intravenous antibiotics between subjects randomized to HS and IS
during the 48 week treatment period
Secondary Outcomes (6)
Symptoms by parent home questionnaire administered weekly
during the 48 week treatment period
Health-related quality of life as assessed by scores from Cystic Fibrosis Questionnaire-Revised Parent Report (CFQ-R), administered quarterly
over the 48 week treatment period
Standardized cough score assessed at study visits
during the 48 week treatment period
Change in weight, height, resting respiratory rate, and room air oxygen saturation
over the 48 week treatment period
Among participants from whom Pseudomonas aeruginosa (Pa) and other CF pathogens were not isolated from respiratory cultures prior to enrollment, the proportion from whom these organisms are isolated from clinically collected respiratory cultures
measured at baseline and at 48 weeks
- +1 more secondary outcomes
Study Arms (2)
Active treatment group
EXPERIMENTAL7% Hypertonic Saline administered via inhalation twice daily for 48 ± 4 weeks
Control group
ACTIVE COMPARATOR0.9% Isotonic Saline administered via inhalation twice daily for 48 ± 4 weeks
Interventions
Administered via inhalation twice daily for 48 ± 4 weeks. The delivery system is a PARI Sprint Junior nebulizer with a PARI Baby face mask or mouthpiece driven by a PARI Pro Neb compressor.
Administered via inhalation twice daily for 48 ± 4 weeks. The delivery system is a PARI Sprint Junior nebulizer with a PARI Baby face mask or mouthpiece driven by a PARI Pro Neb compressor.
Eligibility Criteria
You may qualify if:
- Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND either: (a) A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis or (b) A genotype with two identifiable CF-causing mutations
- Informed consent by parent or legal guardian
- Age 4 months to \< 60 months at Enrollment visit. If participating in Infant Pulmonary Function testing (selected sites), age 4 months to \< 16 months at Enrollment visit.
- Ability to comply with medication use, study visits, and study procedures as judged by the site investigator
You may not qualify if:
- Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 1 week preceding Enrollment visit
- Acute wheezing at Enrollment visit (prior to HS test dose), or at Infant PFT visit (prior to infant pulmonary function testing), as applicable
- Oxygen saturation \< 95% (\< 90% in centers located above 4000 feet elevation) at Enrollment visit (prior to HS test dose) or at Infant PFT visit (prior to infant pulmonary function testing), as applicable
- Other major organ dysfunction, excluding pancreatic dysfunction
- Physical findings that would compromise the safety of the subject or the quality of the study data as determined by the site investigator
- Investigational drug use within 30 days prior to Enrollment visit, or within 30 days prior to Infant PFT visit as applicable
- Treatment with inhaled hypertonic saline at any concentration within 30 days of Enrollment visit, or within 30 days prior to Infant PFT visit as applicable
- Chronic lung disease not related to CF
- Intolerance of test dose of HS at Enrollment visit
- A sibling that has been randomized and is still enrolled in ISIS002
- History of adverse reaction to sedation
- Clinically significant upper airway obstruction as determined by the Site Investigator (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)
- Severe gastroesophageal reflux, defined as persistent frequent emesis despite anti-reflux therapy
- Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 2 weeks preceding visit
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (30)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Stanford University / Lucile S. Packard Children's Hospital
Palo Alto, California, 94304, United States
The Children's Hospital
Aurora, Colorado, 80045, United States
Children's Memorial Hospital and Northwestern University
Chicago, Illinois, 60614, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
University of Louisville
Louisville, Kentucky, 40202, United States
Johns Hopkins University / Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
University of Michigan / C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
Washington University in St. Louis
St Louis, Missouri, 63110, United States
University of Nebraska
Omaha, Nebraska, 68198, United States
Women and Children's Hospital of Buffalo
Buffalo, New York, 14222, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital, Pulmonary Division
Columbus, Ohio, 43205, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15201, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84108, United States
University of Virginia - Pediatric Respiratory Medicine
Charlottesville, Virginia, 22908, United States
Children's Hospital & Regional Medical Center
Seattle, Washington, 98105, United States
University of Wisconsin
Madison, Wisconsin, 53792, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
Related Publications (2)
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: 22610452RESULTSubbarao P, Stanojevic S, Brown M, Jensen R, Rosenfeld M, Davis S, Brumback L, Gustafsson P, Ratjen F. Lung clearance index as an outcome measure for clinical trials in young children with cystic fibrosis. A pilot study using inhaled hypertonic saline. Am J Respir Crit Care Med. 2013 Aug 15;188(4):456-60. doi: 10.1164/rccm.201302-0219OC.
PMID: 23742699DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Stephanie Davis, MD
University of North Carolina, Chapel Hill
- PRINCIPAL INVESTIGATOR
Margaret Rosenfeld, MD, MPH
Children's Hospital and Regional Medical Center
- PRINCIPAL INVESTIGATOR
Felix Ratjen, MD, PhD
University of Toronto Hospital for Sick Children
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
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 1, 2008
First Posted
July 3, 2008
Study Start
April 1, 2009
Primary Completion
October 1, 2011
Study Completion
November 1, 2011
Last Updated
February 12, 2013
Record last verified: 2013-02