Preventive Inhalation of Hypertonic Saline in Infants With Cystic Fibrosis

• Status: Completed
• Trial end date: 2017-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess whether 6% hypertonic saline (HS) is a safe and effective preventive therapy in newborns and infants with cystic fibrosis (CF).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Heidelberg University
• Collaborator: German Center for Lung Research

Criteria

Inclusion Criteria:

1. Confirmed diagnosis of CF established in neonatal period either via CF newborn screening (NBS) or because of symptoms typical for CF (e.g. meconium ileus), positive family history or positive prenatal screening and fulfilling at least one of the following three criteria:

• sweat chloride ≥ 60mEq/L

• two CF causing mutations of CFTR gen

• alterations of transepithelial potential difference of nasal or rectal epithelia typical for CF.

2. Age at enrolment is 0 to 4 months.

3. Patient's and parent's ability to comply with medication use, study visits, and study procedures is judged by the investigator (therefore parents have to understand the character of the study and individual consequences).

4. Participation in this study is voluntary. Only patients, whose parents or legal guardians gave written consent, are included.

Exclusion Criteria:

1. Born < 30 weeks gestation.

2. Prolonged mechanical ventilation in the first 3 months of life.

3. A significant medical disease or condition other than CF likely to interfere with the child's ability to complete the entire protocol.

4. Previous major surgery except for meconium ileus.

5. Other major organ dysfunction, excluding pancreatic or hepatic dysfunction or another condition due to cystic fibrosis.

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

7. History of adverse reaction to sedation.

8. Known hypersensitivity to study treatment.

9. Participation in other interventional studies at the same time.

Criteria, which lead to a displacement of the procedures in sedation until the child has recovered:

• Clinically significant upper airway obstruction as determined by investigator (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnoea).

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

• Oxygen saturation <95% before initial pulmonary function test or initial MRI.

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