Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Diagnostic Test: Lung T1 MRI

• Registration Number: NCT04994301
• Lead Sponsor: University Hospitals Cleveland Medical Center

Brief Summary

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).

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 Timeline

• Study Start: 2020-12-15
• Study First Submitted: 2021-07-29
• Study First Submitted QC: 2021-07-29
• Study First Posted: 2021-08-06
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-30
• Last Update Posted: 2024-02-01
• Primary Completion: 2024-12-31
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

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

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patient with Cystic Fibrosis	Lung T1 MRI	Patients (male, female) age 5-11 with confirmed diagnosis of Cystic Fibrosis. These patients will begin clinically prescribed FDA-approved Trikafta therapy.

Primary Outcome Measures

Name	Time	Method
Lung T1-MRI	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	Evaluate lung T1-MRI (% Normal Lung Perfusion) to assess lung perfusion changes associated with FDA-approved Trikafta therapy in pediatric cystic fibrosis patients.

Secondary Outcome Measures

Name	Time	Method
Spirometry	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	Clinical standard pulmonary function test (FEV1 % Predicted).
Multiple breath washout (MBW)	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	Multiple breath washout to assess lung clearance index (LCI) applied per CF clinical standard.

Trial Locations

Locations (3)

Riley Hospital for Children; 🇺🇸 Indianapolis, Indiana, United States

CS Mott Children's Hospital; 🇺🇸 Ann Arbor, Michigan, United States

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States