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Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis (ISIS Pilot)

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
CF Therapeutics Development Network Coordinating Center
ClinicalTrials.gov Identifier:
NCT00546663
First received: October 17, 2007
Last updated: August 22, 2011
Last verified: August 2011
History of Changes
  Purpose

This is an open-label pilot study of the safety and tolerability of 7% hypertonic saline inhaled twice daily for 14 days in infants with CF, 12 to 30 months of age.


Condition Intervention
Cystic Fibrosis
 Drug: inhaled 7% hypertonic saline (HS)

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study to Evaluate the Tolerability of Inhaled 7% Hypertonic Saline in Infants With Cystic Fibrosis

Resource links provided by NLM:

U.S. FDA Resources

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

Primary Outcome Measures:
  • The proportion of infants who are intolerant of single and repeated doses of HS according to protocol-defined criteria [ Time Frame: At the enrollment visit, during the period of home administration (Days 0 to 14), and at the final study visit. ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • New or increased cough, increased qualitative breathing rate, new or increased noisy breathing, or new or increased emesis at home as noted by parents on the daily symptom report [ Time Frame: During the period of home administration (Days 0 to 14) ] [ Designated as safety issue: Yes ]
  • Change in respiratory rate, oxygen saturation, or cough frequency between the baseline measurement and measurements obtained after HS administration [ Time Frame: At the enrollment visit ] [ Designated as safety issue: Yes ]
  • Change in respiratory rate, oxygen saturation, or cough frequency between the baseline value at the enrollment visit and the value at the final study visit [ Time Frame: Over two weeks of study participation ] [ Designated as safety issue: Yes ]
  • Unanticipated adverse events [ Time Frame: Over two weeks of study participation ] [ Designated as safety issue: Yes ]
  • Adherence, as measured by (1) the number of doses of HS administered per the home symptom report and (2) returned study drug vials [ Time Frame: During the period of home administration (Days 0 to 14) ] [ Designated as safety issue: No ]

Enrollment: 19
Study Start Date: September 2007
Study Completion Date: July 2008
Primary Completion Date: May 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open-label Drug: inhaled 7% hypertonic saline (HS)

7% hypertonic saline administered twice daily for 14 days by nebulization.

A Pari Sprint Junior nebulizer equipped with a Pari Baby face mask and a Pari Proneb compressor will be used to administer the HS (PARI Respiratory Equipment, Inc., Midlothian, VA).

To minimize the risk of cough and bronchospasm with HS inhalation, infants will be pre-treated prior to each dose of HS with albuterol by metered dose inhaler

Other Name: Hyper-Sal™, inhaled saline

Detailed Description:

The efficacy and safety of hypertonic saline (HS) in CF patients over 6 years of age has been demonstrated in clinical trials of 2 to 48 weeks' duration. Based on these results, a large randomized, placebo-controlled trial of the efficacy and safety of 7% HS administered twice daily for 48 weeks to infants with CF, 4 to 15 months of age at enrollment, is planned (the Infant Study of Inhaled Saline (ISIS) trial). It is anticipated that 150 infants at up to 16 sites will be enrolled in the ISIS trial.

To date, the only evaluations of the safety of HS in infants with CF have been small single-dose studies. There has been no evaluation of the tolerability of chronic HS administration. The goal of this study is to assess the safety and tolerability of exposure to 14 days of 7% HS administered twice daily in infants with CF, prior to enrolling subjects in the planned large, randomized, controlled trial. Conduct of this study will provide evidence for the tolerability of chronic HS administration in infants with CF and estimates of the proportion of infants who do not tolerate chronic HS treatment. The results will be used to establish the appropriate measures of tolerability at enrollment in the ISIS trial, and to refine sample size estimates to account for withdrawal due to intolerance of HS.

  Eligibility

Ages Eligible for Study:   12 Months to 30 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of CF as defined by one or more clinical features of CF and a documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two well characterized disease causing mutations
  • Informed consent by parent or legal guardian
  • 12-30 months of age at enrollment

Exclusion Criteria:

  • Wheezing at the baseline evaluation at the enrollment visit
  • Oxygen saturation < 95 % at the baseline evaluation at the enrollment visit
  • Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate, or new rhinorrhea, nasal congestion or rhinorrhea, with onset in the week preceding the enrollment visit
  • Investigational drug use within 30 days prior to the enrollment visit
  • Known intolerance of albuterol
  • Current enrollment in a therapeutic clinical trial
  • Condition or situation which, in the opinion of the investigator, would affect the ability of the patient or family to complete study procedures
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00546663

Locations
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27599-7248
United States, Washington
Children's Hospital and Regional Medical Center
Seattle, Washington, United States, 98105
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: Margaret Rosenfeld, MD, MPH Seattle Children's Hospital
Principal Investigator: Stephanie Davis, MD University of North Carolina
Principal Investigator: Felix Ratjen, MD, PhD The Hospital for Sick Children
  More Information

No publications provided

Responsible Party: CF Therapeutics Development Network Coordinating Center
ClinicalTrials.gov Identifier: NCT00546663     History of Changes
Other Study ID Numbers: ISIS001
Study First Received: October 17, 2007
Last Updated: August 22, 2011
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

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

ClinicalTrials.gov processed this record on May 16, 2013