Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

Cystic Fibrosis Clinical Trial

Official title:

Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04391322
• Other study ID #: 1000063021
• Status: Recruiting
• Start date: October 20, 2020
• Est. completion date: January 2024

Study information

• Verified date: December 2022
• Source: The Hospital for Sick Children
• Contact: Giles Santyr
• Phone: 4168137654
• Email: giles.santyr[@]sickkids.ca
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

In this study, MRI of the lungs of healthy volunteers and participants with cystic fibrosis (stable and participants initiating CFTR modulator treatment) will be performed over a period of 6 months to determine if lung MRI is able detect structural and functional abnormalities/changes in early cystic fibrosis disease. During the 6 month period, 3 study visits will occur. 70 subjects aged 6 and older will participate in this study. Xenon MRI is a non-invasive imaging technique that does not involve x-rays or ionizing radiation. Rather, this imaging method utilizes the same hardware and software principles that are used for conventional proton MRI of patients in a hospital.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 70
• Est. completion date: January 2024
• Est. primary completion date: December 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 6 Years to 100 Years

Eligibility

Inclusion Criteria:

• Participants must be greater than or equal to 6 years of age

• Informed consent by patient or parent/guardian consent and participant assent when appropriate.

• Able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition

Exclusion Criteria:

• Medical instability that would preclude the ability to undergo the required investigations

• FEV1 % predicted < 40%

• Severe claustrophobia

• Does not meet MRI screening criteria

• Cough within the past 3 days prior to study visit

• Usage of oral antibiotics within 3 weeks prior to study visit

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Lung Diseases
• Pulmonary Fibrosis

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
The Hospital for Sick Children

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reproducibility	Establish the interscan reproducibility of MRI over three visits in healthy volunteers and in patients with stable CF lung disease and compare to conventional breathing tests	12 months
Primary	Responsiveness	Assess responsiveness of MRI to treatment effects over three visits in CF patients receiving CFTR-modulator therapy and compare to conventional breathing tests	12 months
Secondary	Correlation	Correlate changes in lung structure and function detected by MRI imaging with clinical outcomes including FEV1 rate of decline, frequency of pulmonary exacerbations, days of IV antibiotics and hospital days to treat pulmonary exacerbation and change in health-related quality of life measures (CFQ-R scores and CF-QUEST) in the setting of patients with stable CF lung disease and those receiving CFTR-modulator therapy.	12 months