RecruitingNCT06216704

Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis


Sponsor

Massachusetts General Hospital

Enrollment

36 participants

Start Date

Apr 1, 2024

Study Type

OBSERVATIONAL

Conditions

Summary

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.


Eligibility

Min Age: 13 YearsMax Age: 20 Years

Inclusion Criteria4

  • -20 years old
  • Cystic fibrosis with pancreatic insufficiency
  • Must have a stable treatment regimen, including CFTR modulator usage unchanged for the prior three months
  • Liver transplant recipients will be eligible, as long as they are at least 1 year post-transplant and are no longer on Prednisone for immunosuppressive therapy

Exclusion Criteria4

  • Diagnosis of other chronic disease affecting bone health
  • Active use (within the past 3 months) of medications that are known to affect skeletal metabolism
  • CF exacerbation or glucocorticoid exposure within the prior 1 month
  • Lung transplant

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Interventions

DIAGNOSTIC_TESTMagnetic resonance relaxometry

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.

DIAGNOSTIC_TESTMagnetic resonance spectroscopy

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.

DIAGNOSTIC_TESTBlood Draw

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.

DIAGNOSTIC_TESTDXA

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.

DIAGNOSTIC_TESTpQCT

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.


Locations(1)

Boston Children's Hospital

Boston, Massachusetts, United States

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NCT06216704


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