Infant Study of Inhaled Saline in Cystic Fibrosis

The purpose of this study is to assess whether 7% hypertonic saline (HS) is an effective and safe therapy in infants and young children with CF.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

CF Therapeutics Development Network Coordinating Center

Collaborator: National Heart, Lung, and Blood Institute (NHLBI)

Stanford Investigator(s):

Intervention(s):

  • Drug: 7% Hypertonic Saline (HS)
  • Drug: 0.9% Isotonic Saline (IS)

Phase:

N/A

Eligibility


Inclusion Criteria:

   - Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND
   either: (a) A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine
   iontophoresis or (b) A genotype with two identifiable CF-causing mutations

   - Informed consent by parent or legal guardian

   - Age 4 months to < 60 months at Enrollment visit. If participating in Infant Pulmonary
   Function testing (selected sites), age 4 months to < 16 months at Enrollment visit.

   - Ability to comply with medication use, study visits, and study procedures as judged by
   the site investigator

Exclusion Criteria:

   - Acute intercurrent respiratory infection, defined as an increase in cough, wheezing,
   or respiratory rate with onset in 1 week preceding Enrollment visit

   - Acute wheezing at Enrollment visit (prior to HS test dose), or at Infant PFT visit
   (prior to infant pulmonary function testing), as applicable

   - Oxygen saturation < 95% (< 90% in centers located above 4000 feet elevation) at
   Enrollment visit (prior to HS test dose) or at Infant PFT visit (prior to infant
   pulmonary function testing), as applicable

   - Other major organ dysfunction, excluding pancreatic dysfunction

   - Physical findings that would compromise the safety of the subject or the quality of
   the study data as determined by the site investigator

   - Investigational drug use within 30 days prior to Enrollment visit, or within 30 days
   prior to Infant PFT visit as applicable

   - Treatment with inhaled hypertonic saline at any concentration within 30 days of
   Enrollment visit, or within 30 days prior to Infant PFT visit as applicable

   - Chronic lung disease not related to CF

   - Intolerance of test dose of HS at Enrollment visit

   - A sibling that has been randomized and is still enrolled in ISIS002

Additional Exclusion Criteria for Participation in Infant Pulmonary Function Testing:

   - History of adverse reaction to sedation

   - Clinically significant upper airway obstruction as determined by the Site Investigator
   (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed
   obstructive sleep apnea)

   - Severe gastroesophageal reflux, defined as persistent frequent emesis despite
   anti-reflux therapy

   - Acute intercurrent respiratory infection, defined as an increase in cough, wheezing,
   or respiratory rate with onset in 2 weeks preceding visit

Ages Eligible for Study

4 Months - 59 Months

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Colleen Dunn
6507360388
Recruiting

Pediatric Trials