Determinants of Early Cystic Fibrosis Lung Disease

NCT03579173 | Completed DMC

• 1 other identifier: 1712581512
• Study Type: observational
• Target: 61
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis in Children

Keywords

Nutritional Status; Infant Pulmonary Function Testing; Lung Clearance Index; Multiple Breath Washout

Outcome Measures

Primary Outcomes (1)

• Lung function

  Forced expiratory volume in 0.5 seconds

  24 months

Secondary Outcomes (2)

• Lung function - FRC

  24 months

• Lung function - MBW

  24 months

Study Arms (3)

Nutrition and 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.

Diagnostic Test: Infant PFT

Nutrition and 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.

Diagnostic Test: Lung Clearance Index

Passive Tidal Breathing and 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.

Diagnostic Test: Infant PFT; Diagnostic Test: Passive tidal breathing

Interventions

Infant PFT DIAGNOSTIC_TEST

Assessed through the raised volume rapid thoracoabdominal compression technique and plethysmography

Nutrition and Infant PFT; Passive Tidal Breathing and Infant PFT

Lung Clearance Index DIAGNOSTIC_TEST

The Lung Clearance Index is derived from the Multiple Breath Washout test. It is the cumulative exhaled volume (the volume of gas needed to wash out resident gas mixture out of the lungs) divided by the FRC. The FRC is the amount of air left in the lungs after normal exhalation.

Nutrition and Lung Clearance Index

Passive tidal breathing DIAGNOSTIC_TEST

During quiet sleep, tidal breathing flow-volume curves are obtained. The tPEF/tE is calculated by taking the time it takes to reach peak expiratory flow divided by the total peak expiratory time.

Passive Tidal Breathing and Infant PFT

Eligibility Criteria

• Age: 1 Month - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Infants and preschoolers with cystic fibrosis diagnosed by newborn screen.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Indiana University: lead

Study Sites (1)

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Study Officials

• Heather Muston, MD

  Indiana University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Clinical Pediatrics

Study Record Dates

• First Submitted: June 25, 2018
• First Posted: July 6, 2018
• Study Start: May 8, 2018
• Primary Completion: February 6, 2019
• Study Completion: February 6, 2019
• Last Updated: June 22, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)