Clinical trial NCT04391322: Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI
Official title: Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Group 1: Healthy Controls
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
Keywords: CFTR modulator treatment
xenon MRI
Lung
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
First posted: 2020-05-18
Enrollment: 86
Sex: ALL
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: This study includes four populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease and 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

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Reproducibility
timeFrame: 36 months
measure: Correlation
timeFrame: 12 months
measure: Responsiveness
timeFrame: 36 months
measure: Younger cohort
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Giles Santyr, PhD
role: PRINCIPAL_INVESTIGATOR
References: