Pain in Fibrous Dysplasia

NCT04125862 | Completed DMC FDA Device

• 1 other identifier: P00030755
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 1

Brief Summary

Pain remains a common and frequently debilitating symptom, particularly during adulthood of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS). For many FD/MAS patients, the amount of pain perceived is not commensurate with the level of detectable musculoskeletal pathology. Using a combination of clinical and biological assessments, this investigation aims to understand what drives pain in FD/MAS.

Conditions

Fibrous Dysplasia/McCune-Albright Syndrome

Outcome Measures

Primary Outcomes (1)

• % signal difference in BOLD signal

  • % signal difference within striatal and limbic network structures during evoked-heat pain fMRI between FD/MAS patients with pain, FD/MAS patients without pain and matched, healthy volunteers.

  50-60 Minutes

Secondary Outcomes (1)

• Numerical clinical pain rating score

  8 weeks

Other Outcomes (1)

• 18F-FDG or 18F-NaF uptake in FD lesion site

  15-30 Minutes

Study Arms (2)

Fibrous Dysplasia/McCune-Albright Syndrome

OTHER

20, Fibrous Dysplasia/McCune-Albright Syndrome Patients with or without pain

Diagnostic Test: MRI-based Neuroimaging; Diagnostic Test: Non-contrast MRI; Diagnostic Test: 18F-FDG-PET/CT; Diagnostic Test: 18F-NaF-PET/CT

Healthy Controls

OTHER

20, matched healthy controls

Diagnostic Test: MRI-based Neuroimaging

Interventions

MRI-based Neuroimaging DIAGNOSTIC_TEST

During evoked pain fMRI, noxious pressure and heat stimuli will be applied in the two anatomical sites noted above. During fMRI data acquisition, a total of 5 pressure stimulations and 5 heat stimulations will be delivered. Both types of pain stimuli will be applied in a 36/17-sec off/on cycle. The off-condition will correspond to a baseline temperature of 35C and the on condition will match the subject-specific and site-specific 7/10 pressure and heat pain thresholds defined during QST procedures. VAS pain ratings associated with evoked pain stimulation will be collected at the end of each scan. The investigators will also explore the structural properties of the CNS by implementing high-resolution anatomical MRI (gray matter volumes) and diffusion tensor imaging (white matter pathway integrity). All CNS imaging will take approximately 50-60 minutes to complete

Fibrous Dysplasia/McCune-Albright Syndrome; Healthy Controls

Non-contrast MRI DIAGNOSTIC_TEST

Musculoskeletal imaging of the affected lower extremity. Patients will be allowed to watch a movie during data acquisition. The following MRI procedures will be completed in order to identify soft tissue lesions in active or painful regions. Fast spin echo (FSE) T2 fat saturated, T1 weighted MRI, FSE proton density MRI, Short-TI Inversion Recovery (STIR), Diffusion weighted MRI (DW-MRI) and 3D Double Echo Steady State (DESS) will be collected. All musculoskeletal MRI will take approximately 30 minutes to complete.

Fibrous Dysplasia/McCune-Albright Syndrome

18F-FDG-PET/CT DIAGNOSTIC_TEST

Data will be collected 30 min after the intravenous administration of 18F-FDG with an expected average dose of approximately 200 MBq. CT data will be collected at a low-dose to minimize radiation exposure. Patients will be positioned supine head-first with arms on the side. The investigators project whole body data to be collected, but in some cases, the field of view may be limited to the patient's skull to below the knees. The data acquisition period is expected to last between 15-30 minutes and total radiation dose will be \~3-4 mSv.

Fibrous Dysplasia/McCune-Albright Syndrome

18F-NaF-PET/CT DIAGNOSTIC_TEST

Data will be collected 30 min after the intravenous administration of 18F-NaF with an expected average dose of approximately 200 MBq. CT data will be collected at a low-dose to minimize radiation exposure. Patients will be positioned supine head-first with arms on the side. The investigators project whole body data to be collected, but in some cases, the field of view may be limited to the patient's skull to below the knees. The data acquisition period is expected to last between 15-30 minutes and total radiation dose will be \~3-4 mSv.

Fibrous Dysplasia/McCune-Albright Syndrome

Eligibility Criteria

• Age: 10 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male and Female subjects
• years of age
• English Speaking ability sufficient to comprehend consent (with parental assistance if minor)
• Diagnosis of Fibrous Dysplasia

You may not qualify if:

• Younger than 10 or older than 45 years old
• Weight \> 285 lbs (weight limit of the MRI table and \< 36lbs)
• Surgery leaving implanted material
• Contraindications to MRI scanning and PET scanning (including presence of a cardiac pacemaker or pacemaker wires, metallic particles in the body, vascular clips in the head or previous neurosurgery, prosthetic heart valves, claustrophobia, previous significant research related exposure to ionizing radiation)
• Same as for the patients, with the addition of the following:
• Use of recreational or illicit drugs History of chronic pain

Sponsors & Collaborators

• Boston Children's Hospital: lead
• National Institutes of Health (NIH): collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Fibrous Dysplasia of Bone; Fibrous Dysplasia, Polyostotic

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: Individual performing data analyses will be blinded to whether dataset corresponds to FD/MAS patient or matched healthy control
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor, BCH

Model Details: 2 parallel cohorts (FD/MAS patients and matched, healthy controls)

Study Record Dates

• First Submitted: August 12, 2019
• First Posted: October 14, 2019
• Study Start: January 5, 2021
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: December 11, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Individual data will start to become available 12/2019 and last for 1.5 years.
• Access Criteria: A two-way material transfer agreement

Locations

US (1)