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Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Observational
Eligible Ages	5 Years - 11 Years
Gender	All

More Inclusion & Exclusion Criteria

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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04994301
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	University Hospitals Cleveland Medical Center
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Chris Flask, PhD
Principal Investigator Affiliation	Case Western Reserve University
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Cystic Fibrosis

Additional Details

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.

Arms & Interventions

Arms

: 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.

Interventions

Diagnostic Test: - Lung T1 MRI

Evaluation of lung T1 MRI to Assess Lung Disease

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Riley Hospital for Children, Indianapolis, Indiana

Status

Recruiting

Address

Riley Hospital for Children

Indianapolis, Indiana, 46202

Site Contact

Clement Ren, MD, MBA, ATSF

[email protected]

317-948-7180

CS Mott Children's Hospital, Ann Arbor, Michigan

Status