Regional Phenotyping of CF and Non-CF Bronchiectasis

NCT04793867 | Recruiting FDA Drug

• 1 other identifier: 2020-0814
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of developing markers of CF lung disease severity, progression, and therapy response. The Investigator's central hypothesis is that image-based markers can forecast pathophysiology prior to spirometric changes.

Conditions

Cystic Fibrosis; Non-CF Bronchiectasis

Keywords

Cystic Fibrosis; hyperpolarized xenon; LCI; Non-CF Bronchiectasis

Outcome Measures

Primary Outcomes (1)

• Correlation of ventilated volume predictions obtained with 129Xe MRI vs 1H MRI

  Quantify the measurement agreement between the ventilated volume of CF and Non-CF Bronchiectasis lungs vs healthy lungs

  Annually for 5 years

Study Arms (2)

CF and Non-CF Bronchiectasis

In the first arm (Aims 1 \& 2) Up to 50 subjects will be recruited-approximately 25 with normal FEV1 (\>85% predicted) and 25 with mild to moderate disease. All subjects will be asked to undergo longitudinal (i.e., approximately annually) 129Xe and UTE MRI, spirometry, and lung clearance index (LCI) measurement.

Drug: Xenon

Healthy Subjects

Up to 50 age and sex matched control subjects (i.e., subjects with no known cardiopulmonary disorders) may also be recruited to provide a reference data set from healthy subjects for comparison.

Drug: Xenon

Interventions

Xenon DRUG

Subjects will be scanned on a commercial 3T whole-body MRI scanner equipped with high-performance gradient systems. Natural isotopic abundance or isotopically-enriched xenon gas (\~86% 129Xe, Linde Inc.) will be used for all studies. Subjects will be positioned supine in the scanner with a 129Xe RF coil around their chests. Using conventional proton MRI and a breath-hold acquisition, "scout" images will be obtained to localize the subject for subsequent 129Xe acquisitions. 129Xe images will be acquired using the same breath-hold maneuver and a pulse sequence, covering the entire lung (acquisition time \<10 s, approximately 3-mm in-plane resolution, 15-mm slice thickness or less). Additional scans (sequences) may be performed. A maximum of 4 doses of 129Xe will be given throughout the study following the calibration dose.

Also known as: 129Xe

CF and Non-CF Bronchiectasis; Healthy Subjects

Eligibility Criteria

• Age: 5 Years - 100 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

CF and Non-CF Bronchiectasis patients

You may qualify if:

• CF Patients: Diagnosis of CF based on sweat chloride \>60 mMol/l
• Presence of two disease causing CFTR mutations, or end organ manifestations of disease.
• Age minimum 5 years.
• Care provided by the CCHMC CF Care Center or other regional CF Care Centers if required to achieve recruitment goals.

You may not qualify if:

• Pregnancy or lactation.
• Subjects 5 years of age and older with no known history of cardiopulmonary disease.
• Pregnancy or lactation.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Penny New

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Xenon

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

Intervention Hierarchy (Ancestors)

Noble Gases; Elements; Inorganic Chemicals; Gases

Study Officials

• Zackary I Cleveland, PhD

  CCHMC

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Penny A New, BBA

CONTACT

513-332-7931; penny.new@cchmc.org

Carrie Stevens, BS

CONTACT

513-636-9973; carrie.stevens@cchmc.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 23, 2021
• First Posted: March 11, 2021
• Study Start: February 8, 2021
• Primary Completion: March 31, 2026
• Study Completion (Estimated): March 31, 2027
• Last Updated: February 20, 2025

Record last verified: 2025-02

Locations

US (1)