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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	Yes
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Observational
Eligible Ages	4 Years - 18 Years
Gender	All

More Inclusion & Exclusion 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

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04391322
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	The Hospital for Sick Children
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Giles Santyr, PhD
Principal Investigator Affiliation	The Hospital for Sick Children
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	Canada
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	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

Arms & Interventions

Arms

: 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.

Interventions

Contact a Trial Team

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