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

October 17, 2007
August 22, 2011
September 2007
May 2008   (final data collection date for primary outcome measure)
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 ]
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. ]
Complete list of historical versions of study NCT00546663 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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) ]
  • 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 ]
  • 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 ]
  • Unanticipated adverse events [ Time Frame: Over two weeks of study participation ]
  • 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) ]
Not Provided
Not Provided
 
Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis
A Pilot Study to Evaluate the Tolerability of Inhaled 7% Hypertonic Saline in Infants With Cystic Fibrosis

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.

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.

Interventional
Not Provided
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Cystic Fibrosis
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
Experimental: Open-label
Intervention: Drug: inhaled 7% hypertonic saline (HS)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
19
July 2008
May 2008   (final data collection date for primary outcome measure)

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
Both
12 Months to 30 Months
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada
 
NCT00546663
ISIS001
Yes
CF Therapeutics Development Network Coordinating Center
CF Therapeutics Development Network Coordinating Center
Cystic Fibrosis Foundation
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
CF Therapeutics Development Network Coordinating Center
August 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP