Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

Lung T1 MRI Assessments of Children with CF Initiating Trikafta Therapy

In this observational study, the investigators evaluate the sensitivity of T1-MRI to identify lung perfusion changes in pediatric patients with CF (age = 6-11) before and after initiating FDA-approved Trikafta therapy. The investigators compare these Lung T1 MRI assessments (% Normal lung perfusion) to currently best-available assessments of lung function in CF patients (i.e., MBW (LCI( and Spirometry (FEV1 % Predicted).

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Diagnostic test: Lung T1 MRI

Detailed Description

This is a prospective study with 3 study visits to evaluate the utility of Magnetic Resonance Imaging (MRI) and clinical lung function assessments to detect changes in Cystic Fibrosis (CF) patients before and after administration of the FDA-approved Trikafta therapy. The 3 study visits include:

Visit 1: Before starting Trikafta Visit 2: 3 months from start of Trikafta Visit 3: 6 months from start of Trikafta

Along with the clinical assessments (MBW and Spirometry), all participants will undergo an MRI scan of the lungs to generate quantitative lung T1 maps. The investigators will compare the lung T1 MRI (% Normal Lung Perfusion) to Multiple Breath Washout (LCI) and spirometry (FEV1 % Predicted) as methods to assess lung changes with administration of Trikafta. The investigators will obtain additional clinical assessments from participant's medical records.

This is a multi-site study involving 3 sites.

• Study Type: Observational
• Enrollment (Actual): 56

Contacts and Locations

Study Locations

• United States
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • CS Mott Children's Hospital
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 11 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved

Description

Inclusion Criteria:

• Male or female individuals with a diagnosis of cystic fibrosis and have at least one copy of the F508del mutation.

Exclusion Criteria:

• Subject who cannot hold their breath for up to 15 seconds.
• Subjects who are pregnant.
• Subjects with MRI contraindication (e.g., heart pacemaker, heart defibrillator, metal in within the body.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patient with Cystic Fibrosis; Patients (male, female) age 5-11 with confirmed diagnosis of Cystic Fibrosis. These patients will begin clinically prescribed FDA-approved Trikafta therapy.	Diagnostic test: Lung T1 MRI; Evaluation of lung T1 MRI to Assess Lung Disease

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung T1-MRI	Evaluate lung T1-MRI (% Normal Lung Perfusion) to assess lung perfusion changes associated with FDA-approved Trikafta therapy in pediatric cystic fibrosis patients.	Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Spirometry	Clinical standard pulmonary function test (FEV1 % Predicted).	Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta
Multiple breath washout (MBW)	Multiple breath washout to assess lung clearance index (LCI) applied per CF clinical standard.	Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) after start of Trikafta, Visit 3: 6 months (+/- 30 days) after start of Trikafta

Collaborators and Investigators

• Sponsor: University Hospitals Cleveland Medical Center
• Investigators: Principal Investigator: Chris Flask, PhD, Case Western Reserve University

Study record dates

Study Major Dates

• Study Start (Actual): December 15, 2020
• Primary Completion (Actual): December 15, 2023
• Study Completion (Actual): December 15, 2023

Study Registration Dates

• First Submitted: July 29, 2021
• First Submitted That Met QC Criteria: July 29, 2021
• First Posted (Actual): August 6, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 24, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; Conductance; Transmembrane; Regulator
• Additional Relevant MeSH Terms: Pathologic Processes; Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: STUDY20200689

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Unsure if individual data will need to be presented.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No