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In this observational study, the investigators evaluate the sensitivity of T1-MRI to identify lung perfusion changes in pediatric patients with CF (age = 6-11) before and after initiating FDA-approved Trikafta therapy. The investigators compare these Lung T1 MRI assessments (% Normal lung perfusion) to currently best-available assessments of lung function in CF patients (i.e., MBW (LCI( and Spirometry (FEV1 % Predicted).
This is a prospective study with 3 study visits to evaluate the utility of Magnetic Resonance Imaging (MRI) and clinical lung function assessments to detect changes in Cystic Fibrosis (CF) patients before and after administration of the FDA-approved Trikafta therapy. The 3 study visits include:
Visit 1: Before starting Trikafta Visit 2: 3 months from start of Trikafta Visit 3: 6 months from start of Trikafta
Along with the clinical assessments (MBW and Spirometry), all participants will undergo an MRI scan of the lungs to generate quantitative lung T1 maps. The investigators will compare the lung T1 MRI (% Normal Lung Perfusion) to Multiple Breath Washout (LCI) and spirometry (FEV1 % Predicted) as methods to assess lung changes with administration of Trikafta. The investigators will obtain additional clinical assessments from participant's medical records.
This is a multi-site study involving 3 sites.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patient with Cystic Fibrosis | Patients (male, female) age 5-11 with confirmed diagnosis of Cystic Fibrosis. These patients will begin clinically prescribed FDA-approved Trikafta therapy. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lung T1 MRI | Diagnostic Test | Evaluation of lung T1 MRI to Assess Lung Disease |
|
| Measure | Description | Time Frame |
|---|---|---|
| Lung T1-MRI | Evaluate lung T1-MRI (% Normal Lung Perfusion) to assess lung perfusion changes associated with FDA-approved Trikafta therapy in pediatric cystic fibrosis patients. | Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta |
| Measure | Description | Time Frame |
|---|---|---|
| Spirometry | Clinical standard pulmonary function test (FEV1 % Predicted). | Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta |
| Multiple breath washout (MBW) |
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Inclusion Criteria:
• Male or female individuals with a diagnosis of cystic fibrosis and have at least one copy of the F508del mutation.
Exclusion Criteria:
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Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved
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| Name | Affiliation | Role |
|---|---|---|
| Chris Flask, PhD | Case Western Reserve University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States | ||
| CS Mott Children's Hospital |
Unsure if individual data will need to be presented.
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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Oropharyngeal swab for the extraction of bacterial DNA only.
Multiple breath washout to assess lung clearance index (LCI) applied per CF clinical standard. |
| Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) after start of Trikafta, Visit 3: 6 months (+/- 30 days) after start of Trikafta |
| Ann Arbor |
| Michigan |
| 48109 |
| United States |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |