Saline Hypertonic in Preschoolers



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:3 - 5
Updated:2/1/2019
Start Date:March 1, 2015
End Date:August 31, 2018

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The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice
daily for 48 weeks improves the lung clearance index by multiple breath nitrogen washout in
comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 5)
with cystic fibrosis.

A growing body of evidence supports the importance of intervention in cystic fibrosis (CF)
lung disease during early childhood, in order to potentially delay or prevent irreversible
lung damage. Yet, aside from antimicrobial therapies, the CF community has no clinical trial
evidence base with which to guide chronic pulmonary therapies in preschool children.
Hypertonic saline (HS) is the most attractive chronic maintenance therapy to investigate in
preschool children because it addresses defective mucociliary clearance, an early step in the
cascade of events leading to CF lung disease that is expected to be abnormal prior to the
onset of airway infection and inflammation.

Based on several studies, HS appears to be safe in children less than 6 years of age, but its
effectiveness has been difficult to measure. In a previous study (ISIS), children less than 6
years old receiving HS had the same number of lung infections as children receiving a control
treatment. However, the investigators think that children this young need a more sensitive
test, such as lung function testing, to see if HS works in preventing lung damage. Multiple
Breath Washout (MBW) is a relatively easy lung function test to perform with preschool
children. It calculates a measurement called the Lung Clearance Index (LCI), a sensitive
measure of airway inhomogeneity. In a sub-study of the ISIS study in 25 children, LCI
improved in children that inhaled HS twice daily for 48 weeks.

This is a multicenter, randomized, double-blind, controlled, parallel group trial assessing
LCI in children with CF ages 3 to 5 at enrollment. Participants will be randomized 1:1 to
receive 7% HS (treatment arm) vs. 0.9 % isotonic saline (control arm) administered twice
daily via jet nebulizer for 48 weeks. Study visits will occur at Screening, Enrollment, and
at Weeks 12, 24, 36 and 48. Contact with a parent or legal guardian to assess adherence and
interim medical history will occur at 1, 4 and 8 weeks after enrollment and then quarterly
between subsequent study visits. Except for the screening visit, study visits will occur on a
quarterly basis.

Total duration of participation will be up to 52 weeks. As enrollment will occur over
approximately 18 months, total duration of the study is expected to be up to 30 months (18
months enrollment plus 12 months for the last participants to complete study participation).

Inclusion Criteria:

- Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF
phenotype or positive CF newborn screen AND one or more of the following criteria:

- A documented sweat chloride ≥ 60 milliequivalents of solute per litre (mEq/L) by
quantitative pilocarpine iontophoresis (QPIT)

- A documented genotype with two disease-causing mutations in the Cystic Fibrosis
Transmembrane Conductance Regulator (CFTR) gene

- Informed consent by parent or legal guardian

- Age ≥ 36 months and ≤72 months at Screening visit

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

- Ability to perform technically acceptable MBW measurements at the screening and
enrollment visits

Exclusion Criteria:

- Acute intercurrent respiratory infection, defined as an increase in cough, wheezing,
or respiratory rate with onset within 3 weeks preceding Screening or Enrollment visit

- Acute wheezing at Screening or Enrollment visit

- Oxygen saturation < 95% (<90% in centers located above 4000 feet elevation) at
Screening or Enrollment visit

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

- Investigational drug use within 30 days prior to Screening or Enrollment visit

- Treatment with inhaled hypertonic saline at any concentration within 30 days prior to
Screening or Enrollment visit

- Chronic lung disease not related to CF

- Inability to tolerate first dose of study treatment at the Enrollment visit
We found this trial at
24
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Chapel Hill, North Carolina 27599
(919) 962-2211
Phone: 919-966-1055
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Phone: 513-803-7429
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Phone: 614-722-4766
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Phone: 816-701-5239
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Phone: 412-692-8929
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Phone: 720-777-2945
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Baltimore, Maryland 21287
Phone: 410-614-1804
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Phone: 617-355-5553
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Buffalo, New York 14222
Phone: 716-878-7343
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Phone: 312-227-6266
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Houston, Texas 77030
Phone: 832-822-3946
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Indianapolis, Indiana 46202
Phone: 317-948-7734
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Iowa City, Iowa 52242
Phone: 319-384-7546
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Phone: 414-266-6986
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Minneapolis, Minnesota 55404
Phone: 612-813-6347
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Phone: 407-650-7966
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Palo Alto, California 94304
Phone: 650-721-1132
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Philadelphia, Pennsylvania 19104
Phone: 267-426-9626
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Portland, Oregon 97227
Phone: 503-494-7807
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660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Phone: 314-286-1781
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Phone: 206-987-3317
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Vancouver, British Columbia
Phone: 604-875-2345
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