Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

NCT04994301 | Completed

• 1 other identifier: STUDY20200689
• Study Type: observational
• Target: 56
• Locations: 1 country
• Sites: 3

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).

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Transmembrane; Conductance; Regulator

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• 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

• 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

Study Arms (1)

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

Interventions

Lung T1 MRI DIAGNOSTIC_TEST

Evaluation of lung T1 MRI to Assess Lung Disease

Patient with Cystic Fibrosis

Eligibility Criteria

• Age: 5 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Hospitals Cleveland Medical Center: lead

Study Sites (3)

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

CS Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Biospecimen

Retention: NONE RETAINED

Oropharyngeal swab for the extraction of bacterial DNA only.

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

• Chris Flask, PhD

  Case Western Reserve University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Senior Staff Physician, Adult Cystic Fibrosis Center

Study Record Dates

• First Submitted: July 29, 2021
• First Posted: August 6, 2021
• Study Start: December 15, 2020
• Primary Completion: December 15, 2023
• Study Completion: December 15, 2023
• Last Updated: February 27, 2025

Record last verified: 2025-02

Locations

US (3)