An observational study in Cystic Fibrosis, sponsored by Massachusetts General Hospital. Recruiting at 1 site in United States. Open to participants aged 13 Years to 20 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-10-30.
Sponsored by Massachusetts General Hospital · Observational
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to healthy, matched controls. The investigators will also be assessing their bone mineral density via other imaging modalities, including dual-energy X-ray absorptiometry (DXA) and peripheral quantitative computed tomography (pQCT). This longitudinal project will focus on abnormalities in bone marrow composition, and specifically whether adolescents with diagnosed with CF exhibit increased bone marrow fat, its association with bone mineral density (BMD) and the underlying pathophysiology, including glycemic control, inflammation, and bone turnover markers.
Less than optimal bone health has been seen in children that have cystic fibrosis (CF). This can present as low bone density or altered bone structure, weakening the bones and increasing fragility and fracture risk. As adolescence is especially important in bone development, conditions such as CF during this time can lead to long term bone issues. The underlying mechanisms are not well understood, but what is known is that red bone marrow converts to fat-rich yellow marrow. This study aims to focus on abnormalities in bone marrow, and specifically whether adolescents who have been diagnosed with CF have more bone marrow fat.
The primary hypothesis is that patients with CF will have associated increased fat levels in bone marrow, which will be associated with decreased bone formation and suboptimal bone health. The central objective is to obtain longitudinal data on the differences in bone marrow between patients with CF versus healthy adolescents. Long term, the investigators want to study how abnormal marrow fat and suboptimal bone health relate to one another.
The study involves 36 adolescents diagnosed with CF and 36 matched healthy controls. Eligibility criteria include no other chronic diseases that affect bone health and limited use of bone altering medications in the prior three months. The adolescents with CF will be matched with healthy adolescents based on sex, ancestry, age, and pubertal stage. Additional data on participants with CF will be collected via a chart review that will enable us to more fully characterize their CF.
Imaging will include: MRI of the knee with quantitative marrow fat assessment; dual-energy X-ray absorptiometry (DXA); and peripheral quantitative computed tomography (pQCT). All scans will be for research purposes only. The MRIs will be evaluated for any incidental findings, and if any identified, it will be reported to their primary care physician.
Additionally, blood draws will be used to assess markers of bone formation/resorption and inflammation. In participants with CF, they will have a continuous glucose monitor to assess dysglycemia. All participants will also complete questionnaires.
There will be a baseline visit, and then a follow up visit 1 year later, with identical study procedures at both visits.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 36 is below the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Massachusetts General Hospital is the lead sponsor of 2,536 studies on the registry; 446 are open to participants now.
Of its 214 completed or terminated interventional studies of FDA-regulated products, 161 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
The experimental group will be adolescents aged 13-20 with CF.
The control group will be matched for sex, ancestry, age (within 2 years), and pubertal stage (based on Tanner staging, ± 1 Tanner stage).
Exclusion Criteria:
This group will be 36 adolescents, ages 13-20 years old, who have been diagnosed with cystic fibrosis. All participants will have a two study visits approximately one year apart during which the listed diagnostic testing will be performed.
Diagnostic Test: Magnetic resonance relaxometry · Diagnostic Test: Magnetic resonance spectroscopy · Diagnostic Test: Blood Draw · Diagnostic Test: DXA · Diagnostic Test: pQCT
Controls will be matched for age, Tanner staging, BMI percentile, and ancestry. All participants will have a two study visits approximately one year apart during which the listed diagnostic testing will be performed.
Diagnostic Test: Magnetic resonance relaxometry · Diagnostic Test: Magnetic resonance spectroscopy · Diagnostic Test: Blood Draw · Diagnostic Test: DXA · Diagnostic Test: pQCT
Spin-lattice relaxation (T1) relaxometry acquisition consisting of fast spin echo (FSE) acquisitions through the knee. T1 maps from the T1 relaxometry images will be generated using a two-parameter-fit iterative algorithm developed in-house using IDL software (Harris Geospatial Solutions, Melbourne, FL, USA). Mean T1 values for each region will be recorded. The anatomical locations of these regions will be consistent in size for all subjects and location. The locations chosen for the primary endpoints are ones that are known to be rich in red and yellow marrow, respectively.
Magnetic resonance spectroscopy. MRS will be performed within a 1 mL voxel situated in the medial aspect of the distal femoral metaphysis. A single voxel point resolved spectral acquisition (PRESS) technique will be used to acquire non-water suppressed spectra at multiple echo times. Spectral fits using JMRUI MRS processing software (www.jmrui.eu) to the water and methylene/methyl resonances will be used to quantify peak areas and establish T2 corrected fat/(fat + water) ratios.
Blood draw. Blood draws will be used to attain and assess markers of bone formation/resorption and inflammation. Specific markers of bone formation that will be assessed include osteocalcin (OC) and procollagen type 1 N-terminal propeptide (P1NP), and a marker of bone resorption, c-telopeptide (CTX). Additionally, in participants with CF, we will assess inflammation, with a c-reactive protein (CRP), and dysglycemia, with a continuous glucose monitor.
DXA will be utilized to obtain BMD of the total body, lumbar spine, and hip using a Hologic Horizon densitometer (Hologic Inc, Bedford, MA). Body composition will be obtained from total body scans.
pQCT will be utilized to obtain volumetric BMD (mg/cm3) of the left tibia. Measurements using a Stratec XCT 3000 device (Orthometrix, White Plains, NY) will be obtained at multiple locations, in relation to distal growth plate.
Bone marrow adiposity by magnetic resonance relaxometry (MR relaxometry)
Change in bone marrow adiposity measured by MR relaxometry
Time frame: Baseline and One Year follow-up
Bone marrow adiposity by magnetic resonance spectroscopy (MRS)
Change in bone marrow adiposity measured by MRS
Time frame: Baseline and One Year follow-up
Total body bone mineral density Z-score by Dual-energy X-ray absorptiometry (DXA)
Change in total body less head BMD Z-score
Time frame: Baseline and One Year follow-up
Spine BMD Z-score by DXA
Change in lumbar spine BMD Z-score
Time frame: Baseline and One Year follow-up
Hip BMD Z-score by DXA
Change in hip BMD Z-score
Time frame: Baseline and One year follow-up
Volumetric bone mineral density (vBMD)
Change in quantitative computed tomography (pQCT) scans will be obtained at the left tibia
Time frame: Baseline and One Year follow-up
polar strength strain index
Change in pQCT bone strength measure
Time frame: Baseline and One Year follow-up
osteocalcin
Change in bone formation assessed by osteocalcin (ng/mL)
Time frame: Baseline and One Year follow-up
procollagen type 1 N-terminal propeptide
Change in bone formation assessed by procollagen type 1 N-terminal propeptide (ng/mL)
Time frame: Baseline and One Year follow-up
c-telopeptide
Change in bone resorption assessed by c-telopeptide (pg/ml)
Time frame: Baseline and One Year follow-up
Plan to share: No
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Massachusetts General Hospital