Determinants of Early Cystic Fibrosis Lung Disease

Completed

• Conditions: Cystic Fibrosis in Children
• Interventions: Diagnostic Test: Infant PFT; Diagnostic Test: Lung Clearance Index; Diagnostic Test: Passive tidal breathing

• Registration Number: NCT03579173
• Lead Sponsor: Indiana University

Brief Summary

The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening.

Detailed Description

The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening. We hypothesize that respiratory and nutritional indices during the first year of life are critical determinants of lung function in infants and preschoolers with CF.

Aim #1 - To examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF.

Hypothesis: Infants with CF with poor nutritional status at 6 months of age will have worse lung function, as assessed through the raised volume rapid thoracoabdominal compression technique and plethysmography, compared to those with better nutritional indices, defined as improved weight-for-age and weight-for-length.

Aim #2 - To examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age.

Hypothesis: Infants with CF with poor nutritional indices at 12 months of age will have a higher (worse) LCI at 3-5 years of age compared to those with better nutritional status.

Aim #3 - To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age.

Hypothesis: Abnormalities in passive tidal breathing lung function testing will be associated with abnormal infant pulmonary function testing obtained via the raised volume rapid thoracoabdominal compression technique and plethysmography at 6-12 months of age.

Basic Information
|
Recruitment & Eligibility
|
Study & Design
|
Trial Locations

Study Timeline

• Study Start: 2018-05-08
• Study First Submitted: 2018-06-25
• Study First Submitted QC: 2018-06-25
• Study First Posted: 2018-07-06
• Primary Completion: 2019-02-06
• Study Completion: 2019-02-06
• Status Verified: 2022-06
• Last Update Submitted: 2022-06-16
• Last Update Posted: 2022-06-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 61

Inclusion Criteria

• Informed consent by a parent or legal guardian
• A confirmed diagnosis of CF by newborn screening and either a documented sweat chloride of at least 60 mEq/L by quantitative pilocarpine iontophoresis or a genotype with two CF-causing mutations
• One month to five years of age.

Read More

Exclusion Criteria

• Chronic lung disease not due to CF
• Contraindications to sedation, including structural abnormalities of the upper airway or chest wall and severe gastroesophageal reflux
• Gestational age < 36 weeks

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Passive Tidal Breathing and Infant PFT	Passive tidal breathing	To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age.
Nutrition and Infant PFT	Infant PFT	To examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF.
Nutrition and Lung Clearance Index	Lung Clearance Index	To examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age.
Passive Tidal Breathing and Infant PFT	Infant PFT	To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age.

Primary Outcome Measures

Name	Time	Method
Lung function	24 months	Forced expiratory volume in 0.5 seconds

Secondary Outcome Measures

Name	Time	Method
Lung function - MBW	24 months	Multiple breath washout result
Lung function - FRC	24 months	Functional Residual Capacity

Trial Locations

Locations (1)

Riley Hospital for Children; 🇺🇸 Indianapolis, Indiana, United States