Cystic FibrosisNCT04994301
Evaluation of Lung T1-MRI in Pediatric Cystic Fibrosis Patients
An observational study of Cystic Fibrosis, run by University Hospitals Cleveland Medical Center at 3 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 11 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Lung T1 MRI
- How many people
- 48 participants
- Where
- 3 sitesUnited States
- Study dates
- 15 Dec 2020to 15 Dec 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 11 years
- All sexes
- No healthy volunteers
- Child
Study population: Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved
Who may be able to join
- Male or female individuals with a diagnosis of cystic fibrosis and have at least one copy of the F508del mutation.
Who cannot join
- Subject who cannot hold their breath for up to 15 seconds.
- Subjects who are pregnant.
- Subjects with MRI contraindication (e.g., heart pacemaker, heart defibrillator, metal in within the body.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
CS Mott Children's Hospital
Ann Arbor, Michigan, 48109
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Riley Hospital for Children
Indianapolis, Indiana, 46202
About this study
Official title: Lung T1 MRI Assessments of Children With CF Initiating Trikafta Therapy
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).
Read the detailed description
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.
- Conditions
- Cystic Fibrosis
- Being tested
- Lung T1 MRI
- Keywords
- Cystic Fibrosis, Transmembrane, Conductance, Regulator
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lung T1 MRIDiagnostic test
Evaluation of lung T1 MRI to Assess Lung Disease
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Diagnostic Test: Lung T1 MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Secondary outcomes (2)
Spirometry
Clinical standard pulmonary function test (FEV1 % Predicted).
Measured: 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)
Multiple breath washout to assess lung clearance index (LCI) applied per CF clinical standard.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2021
- Study start15 Dec 2020
- Primary completion15 Dec 2023
- Study completion15 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University Hospitals Cleveland Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chris Flask, PhD, Principal investigator, Case Western Reserve University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04994301
- Sponsor's protocol ID
- STUDY20200689
- Status last verified
- Sep 2026
- First submitted
- 29 Jul 2021
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Unsure if individual data will need to be presented.
Publications
Nasr SZ, Flask CA, Kretzler ME, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Gupta A, Ferrebee M, McBennett KA, Ren CL. Lung T1-MRI and Multi-Breath Washout Detect Longitudinal Changes in Children 6-11 Years Old After Beginning Elexacaftor/Tezacaftor/Ivacaftor (ETI) Therapy. Pediatr Pulmonol. 2026 Apr;61(4):e71628. doi: 10.1002/ppul.71628.
PubMed 42003453Ren CL, Nasr SZ, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Keshock E, Nicholas JL, Ferrebee M, McBennett K, Flask CA. Lung T1 MRI assessments in children with mild cystic fibrosis lung disease. Pediatr Pulmonol. 2024 Oct;59(10):2464-2471. doi: 10.1002/ppul.27039. Epub 2024 May 2.
PubMed 38695557
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lung T1 MRI Assessments of Children With CF Initiating Trikafta Therapy
- ClinicalTrials.gov ID
- NCT04994301
- Acronym / sponsor ID
- STUDY20200689
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Transmembrane, Conductance, Regulator
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- University Hospitals Cleveland Medical CenterUniversity, hospital or other
- Study start
- 15 Dec 2020 (actual)
- Primary completion
- 15 Dec 2023 (actual)
- Study completion
- 15 Dec 2023 (actual)
- First posted
- 6 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient with Cystic Fibrosis | – | Diagnostic Test: Lung T1 MRIPatients (male, female) age 5-11 with confirmed diagnosis of Cystic Fibrosis. These patients will begin clinically prescribed FDA-approved Trikafta therapy. |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved
Key inclusion criteria (1 listed)
- Male or female individuals with a diagnosis of cystic fibrosis and have at least one copy of the F508del mutation.
Key exclusion criteria (3 listed)
- Subject who cannot hold their breath for up to 15 seconds.
- Subjects who are pregnant.
- Subjects with MRI contraindication (e.g., heart pacemaker, heart defibrillator, metal in within the body.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lung T1 MRIEvaluation of lung T1 MRI to Assess Lung Disease | Diagnostic test | Patient with Cystic Fibrosis |
Primary outcome
Lung T1-MRI
Time frame: 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
Evaluate lung T1-MRI (% Normal Lung Perfusion) to assess lung perfusion changes associated with FDA-approved Trikafta therapy in pediatric cystic fibrosis patients.
2 secondary outcomes
Spirometry
Time frame: 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)
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
- Results publication: PubMed 42003453Nasr SZ, Flask CA, Kretzler ME, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Gupta A, Ferrebee M, McBennett KA, Ren CL. Lung T1-MRI and Multi-Breath Washout Detect Longitudinal Changes in Children 6-11 Years Old After Beginning Elexacaftor/Tezacaftor/Ivacaftor (ETI) Therapy. Pediatr Pulmonol. 2026 Apr;61(4):e71628. doi: 10.1002/ppul.71628.
- Results publication: PubMed 38695557Ren CL, Nasr SZ, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Keshock E, Nicholas JL, Ferrebee M, McBennett K, Flask CA. Lung T1 MRI assessments in children with mild cystic fibrosis lung disease. Pediatr Pulmonol. 2024 Oct;59(10):2464-2471. doi: 10.1002/ppul.27039. Epub 2024 May 2.
- Individual participant dataSharing planned: Undecided. Unsure if individual data will need to be presented.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chris Flask, PhD principal investigator
Case Western Reserve University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by University Hospitals Cleveland Medical Center at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lung T1-MRI (time frame: 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04994301, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026; AzTrials last refreshed its copy on 8 Nov 2026.