Clinical trial NCT04994301: Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients
Official title: Lung T1 MRI Assessments of Children with CF Initiating Trikafta Therapy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
Transmembrane
Conductance
Regulator
Lead sponsor: University Hospitals Cleveland Medical Center
Sponsor class: OTHER
First posted: 2021-08-06
Enrollment: 56
Sex: ALL
Minimum age (years): 5
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved

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.

Eligibility 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.

Primary outcomes:
measure: Lung T1-MRI
timeFrame: 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:
measure: Spirometry
timeFrame: 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
measure: Multiple breath washout (MBW)
timeFrame: 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
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Ann Arbor
country: United States
facility: CS Mott Children's Hospital
state: Michigan
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
Study officials:
affiliation: Case Western Reserve University
name: Chris Flask, PhD
role: PRINCIPAL_INVESTIGATOR
References: