Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients

Cystic Fibrosis Clinical Trial

Official title:

Lung T1 MRI Assessments of Children With CF Initiating Trikafta Therapy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04994301
• Other study ID #: STUDY20200689
• Status: Recruiting
• Start date: December 15, 2020
• Est. completion date: December 31, 2024

Study information

• Verified date: January 2024
• Source: University Hospitals Cleveland Medical Center
• Contact: Kimberly M MD, PhD, FACP, MD, PhD
• Phone: 216-844-3267
• Email: Kimberly.McBennett[@]UHhospitals.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 48
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 11 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Diagnostic Test:
• Lung T1 MRI
Evaluation of lung T1 MRI to Assess Lung Disease

Locations

Country	Name	City	State
United States	CS Mott Children's Hospital	Ann Arbor	Michigan
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Riley Hospital for Children	Indianapolis	Indiana

Sponsors (1)

Lead Sponsor	Collaborator
University Hospitals Cleveland Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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