Hypertonic Saline and Mucociliary Clearance in Children

NCT01293084 | Completed Phase 2 Results

• 1 other identifier: CFF Account #LAUBE06A0
• Study Type: interventional
• Target: 17
• Locations: 0 countries
• Sites: N/A

Brief Summary

Previously, the investigators and others have shown that mucociliary clearance (MCC) is defective in patients with cystic fibrosis (CF) and it is now thought that alterations in airway mucus rheology figure prominently in the impairment. Mucociliary clearance works by trapping toxic particles, bacteria and viruses in the lung mucus and then quickly removing the mucus out of the lungs. Defects in MCC typically lead to the accumulation of mucus in the airways, and this in turn is associated with acute infections, chronic bacterial colonization and chronic inflammation. One treatment strategy that is gaining acceptance as an important therapy for improving MCC in adults with CF is the inhalation of the osmotic stimulus, hypertonic saline (HS). A number of studies have shown that acute inhalation of HS (7% saline) significantly improves MCC in adults with CF and results from a recent study indicate that two weeks of inhaling HS leads to a significant increase in MCC that is sustained for 8 hours post inhalation and is associated with significant improvements in FEV1, FVC and FEF25-75 values. Since MCC in patients with CF appears to be impaired by adulthood, any drug that disrupts or slows the impairment in childhood could prove enormously beneficial in the long-term prognosis of the disease. Nevertheless, no studies have been conducted to determine if HS treatment improves MCC in children with CF. This is most problematic for physicians who care for children with CF who have normal FEV1 and FVC values, since it is unclear if they should treat these children with HS or not. This research study is designed to begin to answer this question. The investigators hypothesize that acute inhalation of hypertonic saline (7%) will improve MCC in CF children with normal pulmonary function. Our hypothesis will be tested in a one-year clinical trial that will be randomized and placebo-controlled. Twelve children with CF who are 7-12 years old and have normal FEV1 and FVC values will participate. Our goal will be to compare MCC in these children on two study visits after acute inhalations of placebo (0.12% saline) or hypertonic saline (HS) (7% saline) aerosol. The investigators predict that MCC values after acute inhalation of 7% HS aerosol will be statistically significantly greater than after placebo inhalation.

Conditions

Cystic Fibrosis

Keywords

mucociliary clearance; children; cystic fibrosis; hypertonic saline

Outcome Measures

Primary Outcomes (1)

• Percent Mucociliary Clearance at 60 Minutes

  60 minutes

Secondary Outcomes (1)

• Percent Mucociliary Clearance at 90 Minutes

  90 minutes

Study Arms (2)

7% saline

EXPERIMENTAL

5 mL of 7% saline was inhaled once over a 20 minute period.

Drug: 7% saline

0.12% saline

PLACEBO COMPARATOR

5mL 0.12% saline inhaled once during 20 minutes

Drug: 0.12% saline

Interventions

0.12% saline DRUG

5mL of 0.12% saline inhaled once over 20 minutes

0.12% saline

7% saline DRUG

5mL 7% saline inhaled once over 20 minutes

Also known as: hypertonic saline

7% saline

Eligibility Criteria

• Age: 7 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males and females
• Age 7-12 years old
• Diagnosis of cystic fibrosis by sweat chloride \> 60 meq/L, or presence of two CFTR mutations known to cause CF
• Routinely treated with the short-acting bronchodilator albuterol
• FEV1 \> 90% of predicted values

You may not qualify if:

• FEV1 \< 90% of predicted values
• Routine use of hypertonic saline, mannitol, or amiloride
• Allergic bronchopulmonary aspergillosis (ABPA)
• Sputum colonization with Burkholderia cepacia or multiple antibiotic resistant organisms
• Evidence of a pulmonary exacerbation within past two weeks
• Treated with intravenous or oral antibiotics in the past two weeks for a pulmonary exacerbation
• Presence of an acute respiratory illness characterized by:
• Coughing above baseline values
• Wheezing
• Respiratory distress
• Hemoptysis
• Cannot perform the inhalation maneuvers that are required for drug inhalation or radioaerosol administration

Sponsors & Collaborators

• Johns Hopkins University: lead
• Cystic Fibrosis Foundation: collaborator

Related Publications (1)

• Laube BL, Sharpless G, Carson KA, Kelly A, Mogayzel PJ Jr. Acute inhalation of hypertonic saline does not improve mucociliary clearance in all children with cystic fibrosis. BMC Pulm Med. 2011 Sep 6;11:45. doi: 10.1186/1471-2466-11-45.

  PMID: 21896198 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Sodium Chloride; Saline Solution, Hypertonic

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Hypertonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Beth Laube Professor
• Organization: Johns Hopkins Univeristy

blaube@jhmi.edu

410-502-5791

Study Officials

• Beth L Laube, PhD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 9, 2011
• First Posted: February 10, 2011
• Study Start: July 1, 2007
• Primary Completion: June 1, 2009
• Study Completion: June 1, 2009
• Last Updated: September 22, 2015
• Results First Posted: September 22, 2015

Record last verified: 2015-09