Infant Study of Inhaled Saline in Cystic Fibrosis - Full Text View

Sponsors and Collaborators:	CF Therapeutics Development Network Coordinating Center Cystic Fibrosis Foundation
Information provided by:	CF Therapeutics Development Network Coordinating Center
ClinicalTrials.gov Identifier:	NCT00709280

Purpose

The purpose of this study is to determine whether 7% hypertonic saline (HS) is an effective and safe therapy in infants with CF

Condition	Intervention
Cystic Fibrosis	Drug: 7% Hypertonic Saline (HS) Drug: 0.9% Isotonic Saline (IS)

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Sodium chloride Proline

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Official Title:	Infant Study of Inhaled Saline in Cystic Fibrosis

Further study details as provided by CF Therapeutics Development Network Coordinating Center:

Primary Outcome Measures:

The average change in functional residual capacity (FRC) measured by plethysmography during infant pulmonary function testing (iPFT) [ Time Frame: over the 48 week treatment period ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

The number of protocol-defined pulmonary exacerbations requiring treatment with oral, inhaled or intravenous antibiotics [ Time Frame: over the 48 week treatment period ] [ Designated as safety issue: Yes ]
Rates of adverse events, withdrawal, adherence to treatment, new isolation of CF pathogens from respiratory cultures, resting respiratory rate, and room air oxygen saturations [ Time Frame: over the 48 week treatment period ] [ Designated as safety issue: Yes ]
The rate of protocol-defined intolerance to the test dose of HS [ Time Frame: at the Screening Visit ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	150
Study Start Date:	September 2008
Estimated Study Completion Date:	April 2011
Estimated Primary Completion Date:	December 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental Hypertonic Saline	Drug: 7% Hypertonic Saline (HS) Administered via inhalation twice daily for 48 ± 4 weeks. The delivery system is a PARI Sprint Junior nebulizer with a PARI Baby face mask driven by a PARI Pro Neb compressor.
2: Active Comparator Isotonic Saline	Drug: 0.9% Isotonic Saline (IS) Administered via inhalation twice daily for 48 ± 4 weeks. The delivery system is a PARI Sprint Junior nebulizer with a PARI Baby face mask driven by a PARI Pro Neb compressor.

Detailed Description:

A growing body of evidence supports the importance of intervention in Cystic Fibrosis (CF) lung disease during infancy, in order to potentially delay or prevent irreversible lung disease. Yet, aside from antimicrobial therapies, the CF community has no clinical trial evidence base with which to guide pulmonary therapies in infants and toddlers. Hypertonic Saline (HS) is the most attractive chronic maintenance therapy to investigate in infants 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.

This study is a randomized, parallel group, controlled trial to assess the efficacy and safety of 7% HS inhaled twice daily for 48 weeks among infants with CF 4 to < 16 months of age at enrollment. The primary hypothesis is that, compared to isotonic saline (IS), HS will improve hyperinflation and obstructive lung disease as measured by infant lung function testing. The results of the proposed trial may for the first time provide evidence for early initiation of HS, which, by improving mucociliary clearance, may delay or hinder the cycle of infection and inflammation responsible for progressive airway damage in CF lung disease.

Eligibility

Ages Eligible for Study:	4 Months to 16 Months
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Diagnosis of CF by one or more clinical features of CF AND either: (a) documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis or (b) genotype with two identifiable mutations consistent with CF
Informed consent by parent or legal guardian
4 months to < 16 months of age at Screening Visit
Ability to comply with medication use, study visits, and study procedures as judged by the Site Investigator
Functional residual capacity (FRC) measurement at the Baseline Visit meets acceptability criteria

Exclusion Criteria:

Acute wheezing at Screening Visit (prior to HS test dose) or at Baseline Visit
Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 1 week preceding Screening Visit or 3 weeks preceding Baseline Visit
Oxygen saturation < 95% (< 90% in Denver, Colorado due to altitude) at Screening Visit (prior to HS test dose) or at Baseline Visit
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
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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00709280

Contacts

Contact: Ron Rowbotham, MSc, CCRA

206-884-7550

Ronald.Rowbotham@seattlechildrens.org

Locations

United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, California
Stanford University / Lucile S. Packard Children's Hospital
Palo Alto, California, United States, 94304
United States, Colorado
The Children's Hospital
Aurora, Colorado, United States, 80045
United States, Illinois
Children's Memorial Hospital and Northwestern University
Chicago, Illinois, United States, 60614
United States, Kentucky
University of Louisville
Louisville, Kentucky, United States, 40202
United States, Maryland
Johns Hopkins University / Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Michigan
University of Michigan / C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
United States, Missouri
Washington University in St. Louis
St. Louis, Missouri, United States, 63110
United States, New York
Women and Children's Hospital of Buffalo
Buffalo, New York, United States, 14222
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Nationwide Children's Hospital, Pulmonary Division
Columbus, Ohio, United States, 43205
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Washington
Children's Hospital & Regional Medical Center
Seattle, Washington, United States, 98105
United States, Wisconsin
University of Wisconsin
Madison, Wisconsin, United States, 53792
Canada, Ontario
Hospital For Sick Children
Toronto, Ontario, Canada, M5G 1X8

Sponsors and Collaborators

CF Therapeutics Development Network Coordinating Center

Cystic Fibrosis Foundation

Investigators

Principal Investigator:	Stephanie Davis, MD	The 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

More Information

Responsible Party:	Cystic Fibrosis Foundation Therapeutics, Inc. ( Medical Affairs )
Study ID Numbers:	ISIS002
Study First Received:	July 1, 2008
Last Updated:	July 31, 2008
ClinicalTrials.gov Identifier:	NCT00709280
Health Authority:	United States: Institutional Review Board; Canada: Health Canada; Canada: Ethics Review Committee

Keywords provided by CF Therapeutics Development Network Coordinating Center:

Cystic Fibrosis
hypertonic saline
inhaled saline

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis

Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic fibrosis

Additional relevant MeSH terms:

Pathologic Processes

ClinicalTrials.gov processed this record on January 14, 2009