Infant Study of Inhaled Saline in Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00709280
• Date of registration: 01/07/2008
• Prospective Registration: Yes
• Primary sponsor: CF Therapeutics Development Network Coordinating Center
• Public title: Infant Study of Inhaled Saline in Cystic Fibrosis ISIS
• Scientific title: Infant Study of Inhaled Saline in Cystic Fibrosis
• Date of first enrolment: April 2009
• Target sample size: 321
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00709280
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: N/A

Countries of recruitment

Canada, United States

Contacts

• Name: Margaret Rosenfeld, MD, MPH
• Affiliation: Children's Hospital and Regional Medical Center

• Name: Felix Ratjen, MD, PhD
• Affiliation: University of Toronto Hospital for Sick Children

• Name: Stephanie Davis, MD
• Affiliation: University of North Carolina, Chapel Hill

Key inclusion & exclusion criteria

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



Age minimum: 4 Months
Age maximum: 59 Months
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: 0.9% Isotonic Saline (IS)

Drug: 7% Hypertonic Saline (HS)

Primary Outcome(s)

The rate of protocol-defined pulmonary exacerbations requiring treatment with oral, inhaled or intravenous antibiotics between subjects randomized to HS and IS [Time Frame: during the 48 week treatment period]

Secondary Outcome(s)

Pulmonary function indices measured at baseline and 48 weeks in infants 4 to 15 months of age at enrollment participating in infant pulmonary function testing (N = 100, selected sites) [Time Frame: over the 48 week treatment period]

Among participants from whom Pseudomonas aeruginosa (Pa) and other CF pathogens were not isolated from respiratory cultures prior to enrollment, the proportion from whom these organisms are isolated from clinically collected respiratory cultures [Time Frame: measured at baseline and at 48 weeks]

Health-related quality of life as assessed by scores from Cystic Fibrosis Questionnaire-Revised Parent Report (CFQ-R), administered quarterly [Time Frame: over the 48 week treatment period]

Standardized cough score assessed at study visits [Time Frame: during the 48 week treatment period]

Change in weight, height, resting respiratory rate, and room air oxygen saturation [Time Frame: over the 48 week treatment period]

Symptoms by parent home questionnaire administered weekly [Time Frame: during the 48 week treatment period]

Secondary ID(s)

ISIS002

U01HL092931

U01HL092932

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation Therapeutics

National Heart, Lung, and Blood Institute (NHLBI)