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

Status: Recruiting
Location: See location...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 4
Maximum Age: 18
Healthy Volunteers: t
View:

• 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

Locations
Other Locations
Canada
The Hospital for Sick Children
RECRUITING
Toronto
Contact Information
Primary
Giles Santyr
giles.santyr@sickkids.ca
4168137654
Backup
Sharon Braganza
sharon.braganza@sickkids.ca
4168137654
Time Frame
Start Date: 2020-10-20
Estimated Completion Date: 2025-12
Participants
Target number of participants: 86
Treatments
Group 1
Healthy participants without lung disease
Group 2
Participants with stable cystic fibrosis
Group 3
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
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.
Related Therapeutic Areas
Sponsors
Leads: The Hospital for Sick Children

This content was sourced from clinicaltrials.gov