Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis

NCT ID: NCT00546663

Last Updated: 2011-08-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-09-30
• Study Completion Date: 2008-07-31

Brief Summary

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.

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open-label

Group Type: EXPERIMENTAL

inhaled 7% hypertonic saline (HS)

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

Other Intervention Names

Hyper-Sal™, inhaled saline

Eligibility 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

• Minimum Eligible Age: 12 Months
• Maximum Eligible Age: 30 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

CF Therapeutics Development Network Coordinating Center

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Margaret Rosenfeld, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Stephanie Davis, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina

Felix Ratjen, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

The Hospital for Sick Children

Locations

University of North Carolina

Chapel Hill, North Carolina, United States

Children's Hospital and Regional Medical Center

Seattle, Washington, United States

Hospital for Sick Children

Toronto, Ontario, Canada

Countries

United States; Canada

Other Identifiers

ISIS001

Identifier Type: -

Identifier Source: org_study_id