Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis (ISIS Pilot)
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.
|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|
- 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 ]
- 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 ]
|Study Start Date:||September 2007|
|Study Completion Date:||July 2008|
|Primary Completion Date:||May 2008 (Final data collection date for primary outcome measure)|
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
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.
|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|
|Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|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|