Recruiting

Observational Study

Sponsor:

Massachusetts General Hospital

Code:

NCT06216704

Conditions

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 13 - 20

Healthy Volunteers: Accepted

Interventions

Magnetic resonance relaxometry

Magnetic resonance spectroscopy

Blood Draw

DXA

pQCT

Study Details

Brief 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.

Conditions

Cystic Fibrosis

Study ID

NCT06216704

Start date

Apr 1, 2024

Status verified date

Oct, 2025

Completion date

Jun 30, 2029

Anticipated

Primary completion date

Dec 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 13 - 20

Healthy Volunteers: Accepted

Inclusion Criteria:

  • 13-20 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 Criteria:

  • 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

Study Design

Enrollment

36 participants

Anticipated

Interventions and Outcome Measures

Arms

Cystic Fibrosis

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.

Control

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.

Interventions

Magnetic 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.

Magnetic 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.

Blood 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.

DXA

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

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.

Primary outcome measure

  • Bone marrow adiposity by magnetic resonance relaxometry (MR relaxometry) [ Time Frame: Baseline and One Year follow-up ]
  • Bone marrow adiposity by magnetic resonance spectroscopy (MRS) [ Time Frame: Baseline and One Year follow-up ]

Central Contacts and Locations

Central contacts

Rebecca Gordon, MD

(617) 355-7476rebecca.gordon@childrens.harvard.edu

Locations

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Rebecca Gordon, MD

rebecca.gordon@childrens.harvard.edu

Principal Investigator:

Rebecca Gordon, MD

More Information

Sponsor

Massachusetts General Hospital

Last update posted

Oct 30, 2025

Last verified

Oct, 2025

Keywords

  • Cystic Fibrosis
  • Bone Marrow
  • Dual-energy X-ray absorptiometry
  • Peripheral Quantitative Computed Tomography
  • Magnetic Resonance Imaging
  • pediatrics
  • bone health

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Massachusetts General Hospital on 2025-10-30.