Microbiome Acquisition and Progression of Inflammation and Airway Disease in Infants and Children With Cystic Fibrosis

NCT01661491 | Completed

• 1 other identifier: 1206010476
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

Cystic Fibrosis (CF) is a fatal, recessive genetic disorder characterized by progressive inflammation and lung damage. It is unclear whether current treatment strategies, which focus on detection and eradication of pathogenic microorganisms in the lung, are the best way to prevent the initiation of early inflammation and lung damage. This study asks how early acquisition of microbial flora occurs in infants with CF and healthy baby controls, and whether this process initiates or influences early inflammation and clinical disease progression in CF.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in the Average Unifrac Value in Fecal Microbiome & Metagenome Composition at 4 years

  High throughput sequencing will be used to identify microbial taxa and microbial genes present in feces, and to determine how these change over a period of 4 years

  4 years

Secondary Outcomes (2)

• Change from Baseline in the Amounts of Calprotectin at 4 years

  4 years

• Change from Baseline in the Amounts of Short Chain Fatty Acids at 4 years

  4 years

Study Arms (2)

Cystic Fibrosis

Infants and toddlers with Cystic Fibrosis

Non-cystic fibrosis controls

Infants and toddlers without Cystic Fibrosis

Eligibility Criteria

• Age: 3 Months - 4 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Infants and children up to the age of 4 with Cystic Fibrosis, and age-matched controls without cystic fibrosis will be eligible for this study. Participants will be recruited from the Cystic Fibrosis clinic and Primary Care Clinic of the Yale New Haven Hospital.

You may qualify if:

• laboratory diagnosis of Cystic Fibrosis

You may not qualify if:

• Major organ system disease other than Cystic Fibrosis
• History of prematurity
• Non Cystic Fibrosis control participants:
• Proof of a negative newborn CF screening test
• Major organ system disease
• History of prematurity

Sponsors & Collaborators

• Yale University: lead

Study Sites (1)

Cystic Fibrosis Clinic, Yale New Haven Hospital

New Haven, Connecticut, 06520, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Fecal samples

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Barbara I Kazmierczak, MD PhD

  Yale University

  PRINCIPAL INVESTIGATOR

• Marie Egan, MD

  Yale University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2012
• First Posted: August 9, 2012
• Study Start: August 1, 2012
• Primary Completion: December 1, 2020
• Study Completion: December 1, 2020
• Last Updated: November 13, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)