Fibrous DysplasiaPhase 2NCT05419050
Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
A Phase 2 study testing denosumab in people with Fibrous Dysplasia, run by National Institute of Dental and Craniofacial Research (NIDCR) at 1 site in United States.
Trial summary
Background: Fibrous dysplasia (FD) is a disease that affects the bones. It causes bone lesions that can become weak and lead to fractures, deformity, and nerve injuries. FD bone lesions begin to develop soon after birth and grow during childhood. The lesions stop growing in adults but can still cause disability. Read more about this study
- Who is it for?
- Ages 4 to 14 years
- Conditions the study is for
- Fibrous Dysplasia
- What is being tested?
- denosumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Oct 2022to 9 Jan 2026
- Phase
- Phase 2
- 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 4 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Confirmed diagnosis of fibrous dysplasia
- Age 4 to 14 years
- Concurrent enrollment in the companion Screening and Natural History protocol 98-D-0145
- Provision of signed and dated informed consent form
- Stated willingness of guardian/Legally Authorized Representative (LAR) to comply with all study procedures and availability for the duration of the study
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- Ability of guardian/LAR to understand and the willingness to sign a written informed consent document
- For females of reproductive potential: agreement to use highly effective contraception for during study participation. Highly effective contraception methods include:
- Total abstinence. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
- Combination of the following (a+b or a+c, or b+c):
- Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception
- Placement of an intrauterine device (IUD) or intrauterine system (IUS)
- Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
- For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
- Minimum body weight of 12 kilograms
Who cannot join
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Pregnancy or lactation
- Known allergic reactions to denosumab
- Prior history, or current evidence, of osteomyelitis/osteonecrosis of the jaw
- Planned invasive dental procedure for the course of the study
- Presence of non-healed dental or oral surgery
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- Orthopedic procedure performed less than 6-weeks prior to first day of the denosumab administration (Day 0)
- Acute fracture less than 6-weeks prior to first day of the denosumab administration (Day 0)
- Serum calcium or albumin-adjusted serum calcium below the normal range for the NIH laboratory (patients will be eligible for re-screening after a repletion period lasting up to 6 months)
- 25-hydroxyvitamin D level than 20 ng/mL (patients will be eligible for re screening after a repletion period lasting up to 6 months)
- Untreated or inadequately treated hypophosphatemia as determined by the principal investigator (patients will be eligible for re-screening after initiation or optimization of phosphorus replacement no longer than 6 months)
- Inability to comply with a non-sedated 18F-NaF PET/CT (subjects will be eligible for re- screening after 6 months)
- Use of another investigational agent within the last 3 months prior to the first day of the denosumab administration (Day 0)
- Have any condition which in the opinion of the PI could present a concern for subject safety or difficulty with data interpretation.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
Background:
Fibrous dysplasia (FD) is a disease that affects the bones. It causes bone lesions that can become weak and lead to fractures, deformity, and nerve injuries. FD bone lesions begin to develop soon after birth and grow during childhood. The lesions stop growing in adults but can still cause disability. Researchers want to find ways to stop the growth of FD bone lesions.
Read more
Objective:
To test a study drug (denosumab) in children with FD.
Eligibility:
Children aged 4 to 14 years with FD and who are also enrolled in the Screening and Natural History protocol (98-D-0145).
Design:
Participants will have a screening visit at the NIH clinic or by telehealth. Their medical history will be reviewed.
Participants will stay overnight in the hospital 4 times in 76 weeks. Each stay will last 5 to 7 nights.
Participants will also visit a local lab for blood and urine tests every 4 weeks during the study.
Participants will receive denosumab once every 4 weeks for 48 weeks. The medication is given as a shot injected under the skin using a small needle. Some injections may be performed at home by a caregiver. The caregiver will receive training for this procedure.
Participants will undergo many tests that may be repeated throughout the study. They will have a dental exam. They will have tests of their strength and ability to move freely. They will have x-rays and other scans to get pictures of their bones.
Participants will be given another medicine that is administered through a needle in the arm over 30 minutes.
Read the detailed description
Study Description:
This will be a phase 2, open label, single arm study of denosumab treatment to prevent fibrous dysplasia (FD) lesion progression in children.
Objectives:
Primary Objective:
Evaluate the effect of denosumab on FD lesion progression in children.
Secondary Objectives:
- Evaluate the effects of denosumab on FD lesion activity.
- Evaluate the effect of denosumab on strength and mobility.
- Evaluate the effect of denosumab on pain and quality of life.
- Evaluate the safety and tolerability of denosumab in children with FD.
Endpoints:
Primary Endpoint:
Change in Skeletal Burden Score from baseline to 48 weeks
Secondary Endpoints:
- Percent change in serum bone turnover markers from baseline to 48 weeks: Procollagen 1 Intact N-Terminal Propeptide (P1NP, formation marker), C- telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase
- Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks
- Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax) from baseline to 48 weeks
- Change in functional parameters from baseline to 48 weeks, including muscle strength, range-of-motion, and walking speed
- Change in patient-reported outcome scales evaluating pain and quality of life from baseline to 48 weeks, including PROMIS Pediatric measures of Pain Intensity, Pain Interference, Mobility, and Fatigue.
- Conditions
- Fibrous Dysplasia
- Being tested
- denosumab
- Keywords
- McCune-Albright Syndrome
- 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.
denosumabDrug
monoclonal antibody to receptor activator of nuclear kappa-B ligand (RANKL), a protein involved in regulating osteoclastogenesis
| Study group | Role | What they receive |
|---|---|---|
| treatment | Experimental | treatment arm Receives: Drug: denosumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Skeletal Burden Score
Skeletal Burden Score is a validated measure for quantifying FD disease burden shown to correlate with skeletal outcomes
Measured: 48 weeks
Secondary outcomes (6)
Percent change in serum bone turnover markers from baseline to 48 weeks: procollagen 1 propeptide (P1NP, formation marker), beta crosslaps telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase
reflect underlying bone turnover, and correlate with skeletal outcomes
Measured: 48 weeks
Adverse events
Safety endpoints for expected and unexpected adverse events
Measured: 76 weeks
Change in functional parameters: - Muscle strength - Range-of-motion - Walking speed (6-minute walk)
Outcome measures that reflect activities of daily living
Measured: 48 weeks
Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks
reflect underlying lesion activity and correlate with skeletal outcomes
Measured: 48 weeks
Change in patient-reported outcome scales: - SF10 - Brief Pain Inventory - Brief Fatigue Inventory
Outcome measures to determine pain and quality of life
Measured: 48 weeks
Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax)
reflect underlying lesion activity and correlate with skeletal outcomes
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2022
- Study start12 Oct 2022
- Primary completion9 Jan 2026
- Study completion9 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrous DysplasiaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Alison M Boyce, M.D., Principal investigator, National Institute of Dental and Craniofacial Research (NIDCR)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05419050
- Sponsor's protocol ID
- 10000780
- Other IDs
- 000780-D
- Status last verified
- 15 Jan 2026
- First submitted
- 11 Jun 2022
- Last updated
- 20 Jan 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: all IPD that underlie results in a publication
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
- ClinicalTrials.gov ID
- NCT05419050
- Acronym / sponsor ID
- 10000780
- Conditions
- Fibrous Dysplasia
- Keywords
- McCune-Albright Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)US National Institutes of Health
- Study start
- 12 Oct 2022 (actual)
- Primary completion
- 9 Jan 2026 (actual)
- Study completion
- 9 Jan 2026 (actual)
- First posted
- 15 Jun 2022 (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.
Background:
Fibrous dysplasia (FD) is a disease that affects the bones. It causes bone lesions that can become weak and lead to fractures, deformity, and nerve injuries. FD bone lesions begin to develop soon after birth and grow during childhood. The lesions stop growing in adults but can still cause disability. Researchers want to find ways to stop the growth of FD bone lesions.
Objective:
To test a study drug (denosumab) in children with FD.
Eligibility:
Children aged 4 to 14 years with FD and who are also enrolled in the Screening and Natural History protocol (98-D-0145).
Design:
Participants will have a screening visit at the NIH clinic or by telehealth. Their medical history will be reviewed.
Participants will stay overnight in the hospital 4 times in 76 weeks. Each stay will last 5 to 7 nights.
Participants will also visit a local lab for blood and urine tests every 4 weeks during the study.
Participants will receive denosumab once every 4 weeks for 48 weeks. The medication is given as a shot injected under the skin using a small needle. Some injections may be performed at home by a caregiver. The caregiver will receive training for this procedure.
Participants will undergo many tests that may be repeated throughout the study. They will have a dental exam. They will have tests of their strength and ability to move freely. They will have x-rays and other scans to get pictures of their bones.
Participants will be given another medicine that is administered through a needle in the arm over 30 minutes.
Detailed description
Study Description:
This will be a phase 2, open label, single arm study of denosumab treatment to prevent fibrous dysplasia (FD) lesion progression in children.
Objectives:
Primary Objective:
Evaluate the effect of denosumab on FD lesion progression in children.
Secondary Objectives:
- Evaluate the effects of denosumab on FD lesion activity.
- Evaluate the effect of denosumab on strength and mobility.
- Evaluate the effect of denosumab on pain and quality of life.
- Evaluate the safety and tolerability of denosumab in children with FD.
Endpoints:
Primary Endpoint:
Change in Skeletal Burden Score from baseline to 48 weeks
Secondary Endpoints:
- Percent change in serum bone turnover markers from baseline to 48 weeks: Procollagen 1 Intact N-Terminal Propeptide (P1NP, formation marker), C- telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase
- Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks
- Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax) from baseline to 48 weeks
- Change in functional parameters from baseline to 48 weeks, including muscle strength, range-of-motion, and walking speed
- Change in patient-reported outcome scales evaluating pain and quality of life from baseline to 48 weeks, including PROMIS Pediatric measures of Pain Intensity, Pain Interference, Mobility, and Fatigue.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment | Experimental | Drug: denosumabtreatment arm |
Who may qualify
- Ages
- 4 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Confirmed diagnosis of fibrous dysplasia
- Age 4 to 14 years
- Concurrent enrollment in the companion Screening and Natural History protocol 98-D-0145
- Provision of signed and dated informed consent form
Key exclusion criteria (14 listed)
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Pregnancy or lactation
- Known allergic reactions to denosumab
- Prior history, or current evidence, of osteomyelitis/osteonecrosis of the jaw
- Planned invasive dental procedure for the course of the study
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 |
|---|---|---|
| denosumabmonoclonal antibody to receptor activator of nuclear kappa-B ligand (RANKL), a protein involved in regulating osteoclastogenesis | Drug | treatment |
Primary outcome
Change in Skeletal Burden Score
Time frame: 48 weeks
Skeletal Burden Score is a validated measure for quantifying FD disease burden shown to correlate with skeletal outcomes
6 secondary outcomes
Percent change in serum bone turnover markers from baseline to 48 weeks: procollagen 1 propeptide (P1NP, formation marker), beta crosslaps telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase
Time frame: 48 weeks
Adverse events
Time frame: 76 weeks
Change in functional parameters: - Muscle strength - Range-of-motion - Walking speed (6-minute walk)
Time frame: 48 weeks
Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks
Time frame: 48 weeks
Change in patient-reported outcome scales: - SF10 - Brief Pain Inventory - Brief Fatigue Inventory
Time frame: 48 weeks
Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax)
Time frame: 48 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 20 Jan 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. all IPD that underlie results in a publication
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Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
McCune-Albright Syndrome
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Morbidity in Adults With Craniofacial Fibrous Dysplasia: The CRANIO-DF Study
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Recruiting
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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.
Alison M Boyce, M.D. principal investigator
National Institute of Dental and Craniofacial Research (NIDCR)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing denosumab in people with Fibrous Dysplasia, run by National Institute of Dental and Craniofacial Research (NIDCR) 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 15 inclusion and 14 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 20 Jan 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change in Skeletal Burden Score (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Jan 2026.