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

The Hospital for Sick Children

Status: Enrolling

Conditions: Group 2: Stable CF; Group 3: CF Participants Anticipated to Receive CFTR-modulator Therapy; Group 4: 4-8 yo CF Participants Starting Triple Combination Modulator Therapy; Group 1: Healthy Controls

Study type: Observational

Funder types: Other

Identifiers

NCT04391322

1000063021

Details and patient eligibility

About

This is a prospective, longitudinal observational study conducted at The Hospital for Sick Children (SickKids). The purpose of this study is to evaluate the ability of MRI to detect changes in lung structure and function in CF patients receiving CFTR-modulator therapy. Participants who meet eligibility criteria and consent to the study will undergo study procedures which include spirometry and body plethysmography (optional), multiple breath washout testing, MRI and measurement of quality of life. The study will consist of three to six study visits for Group 1, 2 and 3: baseline, one month (±1 week) and 6 month (±2 week) and if they re-consent then 3 additional visits of 1 year (±6 months). (2 years (±6 months) and 3 years (±6 months) follow-up. Some participants in Group 2 may transition to Group 3.

This study includes three populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease , Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods.

Enrollment: 86 estimated patients

Sex: All

Ages: 4 to 18 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Participants must be greater than or equal to 4 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

Trial design

86 participants in 4 patient groups

Group 1

Description:

Healthy participants without lung disease

Group 2

Description:

Participants with stable cystic fibrosis

Group 3

Description:

Participants with cystic fibrosis, anticipated to receive treatment with CFTR-modulator therapy. Please note: treatment is determined by your physician as part of your normal therapy plan.

Group 4

Description:

Participants with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods.