Fibrous Dysplasia/McCune-Albright SyndromeNCT04125862
Pain in Fibrous Dysplasia
A study testing MRI-based Neuroimaging, Non-contrast MRI and 18F-FDG-PET/CT in people with Fibrous Dysplasia/McCune-Albright Syndrome, run by Boston Children's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Fibrous Dysplasia/McCune-Albright Syndrome
- What is being tested?
- MRI-based Neuroimaging, Non-contrast MRI and 18F-FDG-PET/CT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jan 2021to 1 Dec 2025
- Phase
- Interventional
- 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 10 to 45 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Male and Female subjects
- 10-45 years of age
- English Speaking ability sufficient to comprehend consent (with parental assistance if minor)
- Diagnosis of Fibrous Dysplasia
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: Elucidating Mechanisms of Pain in Adolescent and Adult Fibrous Dysplasia Patients
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.
Read the detailed description
In Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS), healthy bone tissue and marrow is replaced with pre-osteoblastic, fibrous tissue, yielding skeletal deformities and an increased propensity towards fracture, musculoskeletal weakness and bone pain. Despite the frequent use of pharmacological and non-pharmacological analgesic strategies, pain in FD remains common and frequently debilitating, particularly during adulthood. Moreover, for many patients there is a discordance between perceived pain levels and detectable musculoskeletal pathology. To elucidate the mechanism underlying pain in FD/MAS patients, investigators at Boston Children's Hospital (BCH) aim to probe three inter-related domains that are projected to underlie pain experience(s) in FD/MAS patients. These include (i.) the presence of maladaptive central nervous system processes that amplify afferent pain or somatosensory signals, and also facilitate persistent pain; (ii.) aberrant interplay between neurological and musculoskeletal systems; (iii.) a mental health status shaped by the overall burden of living with FD and (iv.) the influence of childhood, FD-related complications on adulthood pain phenotypes. To investigate these four domains hypothesized to underlie FD pain as well as inform on the disconnect between pain and FD disease burden, the investigators will employ methods that complement routine clinical evaluation and diagnostic tests (i.e., 18F-NaF PET/CT or 18F-FDG PET/CT) such as neuroimaging, musculoskeletal MRI and such as neuroimaging, musculoskeletal MRI and assessment of inflammatory and pain mediator expression in blood samples.
- Being tested
- MRI-based Neuroimaging, Non-contrast MRI, 18F-FDG-PET/CT, 18F-NaF-PET/CT
- Medical subject headings
- Fibrous Dysplasia of Bone, Fibrous Dysplasia, Polyostotic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRI-based NeuroimagingDiagnostic 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
Non-contrast MRIDiagnostic 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.
18F-FDG-PET/CTDiagnostic 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.
18F-NaF-PET/CTDiagnostic 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.
| Study group | Role | What they receive |
|---|---|---|
| Fibrous Dysplasia/McCune-Albright Syndrome | Other | 20, Fibrous Dysplasia/McCune-Albright Syndrome Patients with or without pain Receives: 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 Receives: Diagnostic Test: MRI-based Neuroimaging |
What is being measured
The primary outcome is the main question the study is designed to answer.
% 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.
Measured: 50-60 Minutes
Secondary outcomes (1)
Numerical clinical pain rating score
- Numerical clinical pain rating score (NPRS, 0-10 scale) at weeks 0, 1, 4 and 8.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2019
- Study start5 Jan 2021
- Primary completion1 Dec 2025
- Study completion1 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Fibrous Dysplasia/McCune-Albright SyndromeStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 2 parallel cohorts (FD/MAS patients and matched, healthy controls)
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jaymin Upadhyay, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04125862
- Sponsor's protocol ID
- P00030755
- Status last verified
- Oct 2026
- First submitted
- 12 Aug 2019
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Berry C, Boyce AM, Kaban LB, Peacock ZS, Mannstadt M, Upadhyay J; Craniofacial Fibrous Dysplasia Research Team. Identifying Pain Subtypes in Patients With Craniofacial Lesions of Fibrous Dysplasia/McCune-Albright Syndrome. J Oral Maxillofac Surg. 2025 Mar;83(3):366-373. doi: 10.1016/j.joms.2024.12.001. Epub 2024 Dec 4.
PubMed 39710366Golden E, van der Heijden H, Ren B, Randall ET, Drubach LA, Shah N, Cay M, Ebb D, Kaban LB, Peacock ZS, Boyce AM, Mannstadt M, Upadhyay J. Phenotyping Pain in Patients With Fibrous Dysplasia/McCune-Albright Syndrome. J Clin Endocrinol Metab. 2024 Feb 20;109(3):771-782. doi: 10.1210/clinem/dgad589.
PubMed 37804088
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Elucidating Mechanisms of Pain in Adolescent and Adult Fibrous Dysplasia Patients
- ClinicalTrials.gov ID
- NCT04125862
- Acronym / sponsor ID
- P00030755
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- 5 Jan 2021 (actual)
- Primary completion
- 1 Dec 2025 (actual)
- Study completion
- 1 Dec 2025 (actual)
- First posted
- 14 Oct 2019 (actual)
- 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.
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.
Detailed description
In Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS), healthy bone tissue and marrow is replaced with pre-osteoblastic, fibrous tissue, yielding skeletal deformities and an increased propensity towards fracture, musculoskeletal weakness and bone pain. Despite the frequent use of pharmacological and non-pharmacological analgesic strategies, pain in FD remains common and frequently debilitating, particularly during adulthood. Moreover, for many patients there is a discordance between perceived pain levels and detectable musculoskeletal pathology. To elucidate the mechanism underlying pain in FD/MAS patients, investigators at Boston Children's Hospital (BCH) aim to probe three inter-related domains that are projected to underlie pain experience(s) in FD/MAS patients. These include (i.) the presence of maladaptive central nervous system processes that amplify afferent pain or somatosensory signals, and also facilitate persistent pain; (ii.) aberrant interplay between neurological and musculoskeletal systems; (iii.) a mental health status shaped by the overall burden of living with FD and (iv.) the influence of childhood, FD-related complications on adulthood pain phenotypes. To investigate these four domains hypothesized to underlie FD pain as well as inform on the disconnect between pain and FD disease burden, the investigators will employ methods that complement routine clinical evaluation and diagnostic tests (i.e., 18F-NaF PET/CT or 18F-FDG PET/CT) such as neuroimaging, musculoskeletal MRI and such as neuroimaging, musculoskeletal MRI and assessment of inflammatory and pain mediator expression in blood samples.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
- Model description
- 2 parallel cohorts (FD/MAS patients and matched, healthy controls)
| Arm | Type | Receives |
|---|---|---|
| Fibrous Dysplasia/McCune-Albright Syndrome | Other | Diagnostic Test: MRI-based Neuroimaging, Diagnostic Test: Non-contrast MRI, Diagnostic Test: 18F-FDG-PET/CT, Diagnostic Test: 18F-NaF-PET/CT20, Fibrous Dysplasia/McCune-Albright Syndrome Patients with or without pain |
| Healthy Controls | Other | Diagnostic Test: MRI-based Neuroimaging20, matched healthy controls |
Who may qualify
- Ages
- 10 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Male and Female subjects
- 10-45 years of age
- English Speaking ability sufficient to comprehend consent (with parental assistance if minor)
- Diagnosis of Fibrous Dysplasia
Key exclusion criteria (6 listed)
- 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)
- Use of recreational or illicit drugs History of chronic pain
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 |
|---|---|---|
| MRI-based NeuroimagingDuring 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 | Diagnostic test | Fibrous Dysplasia/McCune-Albright Syndrome, Healthy Controls |
| Non-contrast MRIMusculoskeletal 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. | Diagnostic test | Fibrous Dysplasia/McCune-Albright Syndrome |
| 18F-FDG-PET/CTData 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. | Diagnostic test | Fibrous Dysplasia/McCune-Albright Syndrome |
| 18F-NaF-PET/CTData 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. | Diagnostic test | Fibrous Dysplasia/McCune-Albright Syndrome |
Primary outcome
% signal difference in BOLD signal
Time frame: 50-60 Minutes
• % 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.
1 secondary outcome
Numerical clinical pain rating score
Time frame: 8 weeks
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 27 Oct 2026.
- Derived from this study: PubMed 39710366Berry C, Boyce AM, Kaban LB, Peacock ZS, Mannstadt M, Upadhyay J; Craniofacial Fibrous Dysplasia Research Team. Identifying Pain Subtypes in Patients With Craniofacial Lesions of Fibrous Dysplasia/McCune-Albright Syndrome. J Oral Maxillofac Surg. 2025 Mar;83(3):366-373. doi: 10.1016/j.joms.2024.12.001. Epub 2024 Dec 4.
- Derived from this study: PubMed 37804088Golden E, van der Heijden H, Ren B, Randall ET, Drubach LA, Shah N, Cay M, Ebb D, Kaban LB, Peacock ZS, Boyce AM, Mannstadt M, Upadhyay J. Phenotyping Pain in Patients With Fibrous Dysplasia/McCune-Albright Syndrome. J Clin Endocrinol Metab. 2024 Feb 20;109(3):771-782. doi: 10.1210/clinem/dgad589.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MRI-based Neuroimaging, Non-contrast MRI and 18F-FDG-PET/CT in people with Fibrous Dysplasia/McCune-Albright Syndrome, run by Boston Children's Hospital at 1 site 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 4 inclusion and 6 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 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
% signal difference in BOLD signal (time frame: 50-60 Minutes). 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 NCT04125862, which also lists 2 linked publications.
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 27 Oct 2026.