Type 2 Inflammation in Adults With Cystic Fibrosis

NCT07892443 | Not Yet Recruiting

• 2 other identifiers: HSR3038291K23HL183757-01
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this observational study is to better understand why some adults with cystic fibrosis (CF) continue to have repeated pulmonary exacerbations, or episodes of worsening respiratory symptoms, despite treatment with highly effective CFTR modulator therapy. The study will focus on whether a type of inflammation called type 2 (T2) inflammation may contribute to these pulmonary exacerbations. The main questions this study aims to answer are: Are signs of T2 inflammation more common in adults with CF who have frequent pulmonary exacerbations? Are there differences in inflammation in the nose and airways between people who have frequent pulmonary exacerbations and those who do not? How do immune cells and inflammation change during a pulmonary exacerbation? Participants will: Allow researchers to collect information about their CF and pulmonary exacerbations from their medical records. Complete questionnaires about respiratory and sinus symptoms and quality of life. Provide research blood samples, samples from inside the nose, and exhaled breath condensate. Be followed for up to approximately 3 years, with additional research samples collected during qualifying pulmonary exacerbations when feasible. Participants will continue to receive their usual CF medical care. No experimental medications or treatments will be given as part of this study.

Conditions

Cystic Fibrosis (CF); Type 2 Inflammatory Disorders

Keywords

cystic fibrosis; type 2 inflammation

Outcome Measures

Primary Outcomes (1)

• Baseline Stable-State Absolute Peripheral Blood Eosinophil Count

  Absolute peripheral blood eosinophil count measured during clinical stability at baseline. Eosinophil counts will be compared between participants who, after 36 months of prospective observation, are classified as having modulator-refractory CF (average of ≥2 qualifying pulmonary exacerbations per year) or modulator-responsive CF (average of \<2 qualifying pulmonary exacerbations per year).

  Baseline

Secondary Outcomes (3)

• Change in Nasal Epithelial Gene Expression from Baseline to Pulmonary Exacerbation

  Baseline and qualifying pulmonary exacerbations occurring up to Month 36.

• Change in Exhaled Breath Condensate Inflammatory Mediator Concentrations from Baseline to Pulmonary Exacerbation

  Baseline and qualifying pulmonary exacerbations occurring up to Month 36.

• Change in Peripheral Blood Immune Cell Phenotypes from Baseline to Pulmonary Exacerbation

  Baseline and qualifying pulmonary exacerbations occurring up to Month 36.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with cystic fibrosis receiving highly effective CFTR modulator therapy who receive care through the University of Virginia Adult Cystic Fibrosis Program. Participants will be enrolled while clinically stable and followed prospectively for up to approximately 3 years to characterize pulmonary exacerbations and inflammatory phenotypes. After 24 months of prospective observation, participants will be classified according to pulmonary exacerbation frequency for study analyses.

You may qualify if:

• Age 18 years or older
• Diagnosis of cystic fibrosis
• Receiving highly effective CFTR modulator therapy (HEMT) for at least 6 weeks before enrollment
• Clinically stable at the time of enrollment
• Receives cystic fibrosis care through the University of Virginia Adult Cystic Fibrosis Program
• Able and willing to provide informed consent and participate in study procedures

You may not qualify if:

• Active pulmonary exacerbation at the time of enrollment
• Hospitalization for a pulmonary exacerbation within the 4 weeks before enrollment
• New systemic antibiotic treatment for respiratory symptoms within the 4 weeks before enrollment
• Systemic corticosteroid treatment for respiratory symptoms within the 4 weeks before enrollment
• Pregnancy at the time of enrollment
• Any condition that, in the opinion of the investigator, would make participation in the study unsafe or interfere with completion of study procedures

Sponsors & Collaborators

• University of Virginia: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Related Publications (1)

• Somerville L, Borish L, Noth I, Albon D. Modulator-refractory cystic fibrosis: Defining the scope and challenges of an emerging at-risk population. Ther Adv Respir Dis. 2024 Jan-Dec;18:17534666241297877. doi: 10.1177/17534666241297877.

  PMID: 39543951 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood, including plasma, serum, and peripheral blood mononuclear cells (PBMCs); nasal epithelial cells collected by brushing or scraping; nasal lining fluid collected by nasosorption; and exhaled breath condensate.

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

• Lindsay Somerville, MD

  University of Virginia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lindsay Somerville, MD

CONTACT

434-303-1125; LL7Y@uvahealth.org

Larry Borish, MD

CONTACT

LB4M@virginia.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Director of Adult CF Medicine

Study Record Dates

• First Submitted: September 22, 2026
• First Posted: October 23, 2026
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): May 31, 2031
• Study Completion (Estimated): May 31, 2031
• Last Updated: October 23, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ANALYTIC CODE
• Time Frame: De-identified IPD and supporting information will be available beginning after publication of the primary study results and will remain available for at least 5 years following publication.
• Access Criteria: De-identified individual participant data underlying the published results may be made available to qualified researchers with a methodologically sound research proposal. Requests will be reviewed by the Principal Investigator and, as applicable, the University of Virginia. Data will be shared only to the extent permitted by participant consent, IRB requirements, institutional policies, and applicable data-sharing agreements. Requests may be directed to the Principal Investigator.