Cystic Fibrosis Liver DiseaseNCT02979340
MRI to Characterize and Predict CF Liver Disease in PUSH Cohort
An observational study of Cystic Fibrosis Liver Disease and Pancreatic Insufficiency, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 8 sites in United States.
Trial summary
Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis. Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Read more about this study
- Who is it for?
- Ages 6 to 26 years
- Conditions the study is for
- Cystic Fibrosis Liver Disease, Pancreatic Insufficiency
- What is being tested?
- MRI
- Type of treatment
- Surgery or procedure
- How many people
- 115 participants
- Where
- 8 sitesUnited States
- Study dates
- 27 Jan 2017to 27 Dec 2019
- 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 6 to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic Fibrosis and pancreatic insufficiency and enrolled in the CFLD PUSH longitudinal study.
Who may be able to join
- 1. Currently enrolled in longitudinal follow up in PUSH study at a site with MR elastography.
Who cannot join
- Age under 6 years (all current patients at time of opening of this study will be greater than 6 years of age).
- Internal appliance or hardware that is not compatible with MR.
- Inability to obtain MRI within 6 months of US.
- Inability to cooperate with MRI.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins School of Medicine
Baltimore, Maryland, 21217
Cincinnati Children's Hospital Medical
Cincinnati, Ohio, 45229
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Analysis of Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) Cohort Using Advanced MRI to Characterize and Predict CF Liver Disease
Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis.
Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound.
Read more
Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing.
Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone
Read the detailed description
This proposal will be a cross sectional study of the current well-characterized PUSH cohort. Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but Investigators will allow a larger window due to the slow progression of fibrosis).
Blinded radiology review of imaging sequences will be performed by a local study radiologist with central review performed by the Imaging Core. Analysis by the Imaging Core will include calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content for all MRI studies.
- Being tested
- MRI
- Keywords
- Cystic Fibrosis Liver Disease, Pancreatic Insufficiency
- Medical subject headings
- Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIProcedure or surgery
non-invasive assessment of hepatic fibrosis and steatosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change over Time in Liver stiffness and Lipid content over time using MRE
Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.
Measured: BSL, Year 1, Year 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2016 (estimated)
- Study start27 Jan 2017
- Primary completion27 Dec 2019
- Study completion27 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis Liver DiseaseStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The 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
- 115 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Ed Doo, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Averell Sherker, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02979340
- Sponsor's protocol ID
- CFLD-MRE
- Other IDs
- U01DK062456
- Status last verified
- Apr 2025
- First submitted
- 18 Nov 2016
- Last updated
- 11 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Towbin AJ, Ye W, Huang S, Karmazyn BW, Molleston JP, Masand P, Leung DH, Chang S, Narkewicz MR, Alazraki AL, Freeman AJ, Otto RK, Green N, Kamel IR, Karnsakul WW, Magee JC, Tkach J, Palermo JJ. Prospective study of quantitative liver MRI in cystic fibrosis: feasibility and comparison to PUSH cohort ultrasound. Pediatr Radiol. 2023 Oct;53(11):2210-2220. doi: 10.1007/s00247-023-05706-6. Epub 2023 Jul 28.
PubMed 37500799
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Analysis of Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) Cohort Using Advanced MRI to Characterize and Predict CF Liver Disease
- ClinicalTrials.gov ID
- NCT02979340
- Acronym / sponsor ID
- CFLD-MRE
- Keywords
- Cystic Fibrosis Liver Disease, Pancreatic Insufficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 27 Jan 2017 (actual)
- Primary completion
- 27 Dec 2019 (actual)
- Study completion
- 27 Dec 2019 (actual)
- First posted
- 1 Dec 2016 (estimated)
- 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.
Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis.
Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound.
Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing.
Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone
Detailed description
This proposal will be a cross sectional study of the current well-characterized PUSH cohort. Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but Investigators will allow a larger window due to the slow progression of fibrosis).
Blinded radiology review of imaging sequences will be performed by a local study radiologist with central review performed by the Imaging Core. Analysis by the Imaging Core will include calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content for all MRI studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic Fibrosis and pancreatic insufficiency and enrolled in the CFLD PUSH longitudinal study.
Key inclusion criteria (1 listed)
- 1. Currently enrolled in longitudinal follow up in PUSH study at a site with MR elastography.
Key exclusion criteria (4 listed)
- Age under 6 years (all current patients at time of opening of this study will be greater than 6 years of age).
- Internal appliance or hardware that is not compatible with MR.
- Inability to obtain MRI within 6 months of US.
- Inability to cooperate with MRI.
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 |
|---|---|---|
| MRInon-invasive assessment of hepatic fibrosis and steatosis | Procedure or surgery | – |
Primary outcome
Change over Time in Liver stiffness and Lipid content over time using MRE
Time frame: BSL, Year 1, Year 2
Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.
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 11 Apr 2025.
- Background: PubMed 37500799Towbin AJ, Ye W, Huang S, Karmazyn BW, Molleston JP, Masand P, Leung DH, Chang S, Narkewicz MR, Alazraki AL, Freeman AJ, Otto RK, Green N, Kamel IR, Karnsakul WW, Magee JC, Tkach J, Palermo JJ. Prospective study of quantitative liver MRI in cystic fibrosis: feasibility and comparison to PUSH cohort ultrasound. Pediatr Radiol. 2023 Oct;53(11):2210-2220. doi: 10.1007/s00247-023-05706-6. Epub 2023 Jul 28.
- MRE is a study in the ChiLDReN NetworkLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Ed Doo, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Averell Sherker, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
8 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 Liver Disease and Pancreatic Insufficiency, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 8 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 4 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 11 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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?
Change over Time in Liver stiffness and Lipid content over time using MRE (time frame: BSL, Year 1, Year 2). 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 NCT02979340, which also lists 1 linked publication.
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 11 Apr 2025.