Durability of Hypertonic Saline for Enhancing Mucociliary Clearance in Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: sodium chloride (7%)
Intervention description: 4mL nebulized 7% sodium chloride
Phase: Phase 1
Study type: Interventional
Primary purpose: Basic Science
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 16.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: cystic fibrosis; mucociliary clearance; hypertonic saline

Summary:

      Direct measurement of mucociliary and cough clearance (MCC/CC) has been used as a biomarker
      in cystic fibrosis (CF). Additional knowledge of the performance of this biomarker is needed
      to inform exploratory clinical trial design in support of programs to develop new inhaled
      therapies for CF. We hypothesize that MCC/CC measurements can be used to determine the
      durability of action of agents like hypertonic saline (HS) which increase epithelial lining
      fluid height.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Gender: Male or female (non-pregnant, non-lactating)

          -  Cystic fibrosis documented by a compatible clinical and radiographic presentation, and
             sweat chloride > 60 mEq/l or 2 disease causing CFTR mutations.

          -  Severity of Disease:

               1. Must have FEV1 of greater than or equal to 50% of predicted at the screening
                  visit.

               2. Must have an oxygen saturation of >92% on room air as determined by pulse
                  oximetry at the screening visit.

          -  Patient or legally authorized representative agrees to the patient/individual's
             participation in the study by signing and dating the informed consent form after the
             nature of the study has been fully explained and all questions have been
             satisfactorily answered.

        Exclusion Criteria:

          -  Unstable lung disease: As defined by a change in medical regimen during the preceding
             2 weeks; an FEV1 >15% below recent (within 6 months) clinical measurements; or a
             significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse)
             not considered a part of the usual, chronic progression of CF lung disease.

          -  Patients unable or unwilling to be withdrawn from hypertonic saline therapy, dornase
             alfa, or N-acetylcysteine 3 days prior to and for the duration of each Baseline and
             Treatment Period will be excluded.

          -  Patients unable to withhold use of long-acting bronchodilators (i.e., Salmeterol,
             Advair, Formoterol), anti-cholinergics, and vest therapy 12 hours prior to and for the
             duration of each treatment period.

          -  Patients unable to withhold short-acting bronchodilator 6 hours prior to and for the
             duration of each treatment period except as prescribed by the study protocol.

          -  Patients that have received an investigational drug or therapy during the preceding 30
             days.

          -  Patients that have had radiation exposure within the past year that would cause them
             to exceed Federal Regulations by participating in this study.