Fibrous DysplasiaNCT03231644
Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry
An observational study of Fibrous Dysplasia and McCune Albright Syndrome, run by Tovah Burstein at 1 site in United States.
Trial summary
The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients and families to help answer some of the biggest questions about FD/MAS by completing questionnaires about their lives with FD or MAS. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrous Dysplasia, McCune Albright Syndrome, Mazabraud Syndrome
- How many people
- 600 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Oct 2016to Oct 2028
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Community sample of all patients in these particular disease communities.
Who may be able to join
- clinical diagnosis of fibrous dysplasia
- clinical diagnosis of McCune-Albright syndrome
- clinical diagnosis of Mazabraud's syndrome
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Carmel Shemmesh-Rafalowsky contact
United States1 site, 1 recruiting
- Recruiting
Tovah Burstein
Bethesda, Maryland, 20816-2558
Tovah Burstein, MFA, 6033252489, info@fibrousdysplasia.org
About this study
The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients and families to help answer some of the biggest questions about FD/MAS by completing questionnaires about their lives with FD or MAS.
Have you enrolled in the FD/MAS Patient Registry yet? Are you up-to-date on your surveys? Take a trip to www.fdmasregistry.org today to learn more about the project, enroll, complete your surveys, or make sure you aren't due to provide more info!
The FD/MAS Patient Registry: Your story powers research.
Read the detailed description
The FD/MAS Patient Registry is an IRB approved research project that allows patients and families to share their experiences with fibrous dysplasia/McCune-Albright syndrome (FD/MAS) by completing a series of surveys.
The surveys were created in collaboration with patients, parents, clinicians and researchers, so that the data can be used to answer some of the most important questions about FD/MAS, including:
the way the disease develops over time (its "natural history"), the patient experience of the disease, and its impact on quality of life, how and when diagnoses are made, the scope of treatments in use, what surgical techniques work best, and for whom, what other medical interventions work best, and for whom, what social services and therapies are useful, the costs of care to patients and their families, issues that concern patients (such as the impact of pregnancy on FD, or children feeling different and facing stigma), and which research questions and support programs you think are important to fund.
Participation is free and convenient for people with FD/MAS and their legal guardians. You can join today at www.fdmasregistry.org.
- Keywords
- Fibrous Dysplasia, McCune-Albright Syndrome, Mazabrauds, FD/MAS
- 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.
| Study group | Role | What they receive |
|---|---|---|
| FD/MAS Patients | Patients with fibrous dysplasia and/or McCune-Albright syndrome and related disorders. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment satisfaction
FACIT-Treatment Satisfaction scale
Measured: Through study completion, an average of every 2 years
Perceived symptoms of pain
Brief Pain Inventory
Measured: Through study completion, an average of every 2 years
Depression/anxiety
Hospital Anxiety Depression Scale
Measured: Through study completion, an average of every 2 years
Stigma
NeuroQol Pediatric and Adult Stigma short forms
Measured: Through study completion, an average of every 2 years
Health-related Quality of Life
SF-36,PedsQL 4.0
Measured: Through study completion, an average of every 2 years
Financial health
FACIT-Cost
Measured: Through study completion, an average of every 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2017
- Study start31 Oct 2016
- Primary completionOct 2028 (estimated)
- Study completionOct 2028 (estimated)
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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 600 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Tovah Burstein
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Tovah Burstein, Fibrous Dysplasia Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03231644
- Sponsor's protocol ID
- Pro00018980.
- Status last verified
- Aug 2025
- First submitted
- 20 Jul 2017
- Last updated
- 12 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry
- ClinicalTrials.gov ID
- NCT03231644
- Acronym / sponsor ID
- Pro00018980.
- Keywords
- Fibrous Dysplasia, McCune-Albright Syndrome, Mazabrauds, FD/MAS
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 600 participants (estimated)
- Lead sponsor
- Tovah BursteinUniversity, hospital or other
- Study start
- 31 Oct 2016 (actual)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Oct 2028 (estimated)
- First posted
- 27 Jul 2017 (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.
The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients and families to help answer some of the biggest questions about FD/MAS by completing questionnaires about their lives with FD or MAS.
Have you enrolled in the FD/MAS Patient Registry yet? Are you up-to-date on your surveys? Take a trip to www.fdmasregistry.org today to learn more about the project, enroll, complete your surveys, or make sure you aren't due to provide more info!
The FD/MAS Patient Registry: Your story powers research.
Detailed description
The FD/MAS Patient Registry is an IRB approved research project that allows patients and families to share their experiences with fibrous dysplasia/McCune-Albright syndrome (FD/MAS) by completing a series of surveys.
The surveys were created in collaboration with patients, parents, clinicians and researchers, so that the data can be used to answer some of the most important questions about FD/MAS, including:
the way the disease develops over time (its "natural history"), the patient experience of the disease, and its impact on quality of life, how and when diagnoses are made, the scope of treatments in use, what surgical techniques work best, and for whom, what other medical interventions work best, and for whom, what social services and therapies are useful, the costs of care to patients and their families, issues that concern patients (such as the impact of pregnancy on FD, or children feeling different and facing stigma), and which research questions and support programs you think are important to fund.
Participation is free and convenient for people with FD/MAS and their legal guardians. You can join today at www.fdmasregistry.org.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Target follow-up
- 2 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FD/MAS Patients | – | –Patients with fibrous dysplasia and/or McCune-Albright syndrome and related disorders. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Community sample of all patients in these particular disease communities.
Key inclusion criteria (3 listed)
- clinical diagnosis of fibrous dysplasia
- clinical diagnosis of McCune-Albright syndrome
- clinical diagnosis of Mazabraud's syndrome
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Treatment satisfaction
Time frame: Through study completion, an average of every 2 years
FACIT-Treatment Satisfaction scale
Perceived symptoms of pain
Time frame: Through study completion, an average of every 2 years
Brief Pain Inventory
Depression/anxiety
Time frame: Through study completion, an average of every 2 years
Hospital Anxiety Depression Scale
Stigma
Time frame: Through study completion, an average of every 2 years
NeuroQol Pediatric and Adult Stigma short forms
Health-related Quality of Life
Time frame: Through study completion, an average of every 2 years
SF-36,PedsQL 4.0
Financial health
Time frame: Through study completion, an average of every 2 years
FACIT-Cost
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2025.
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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.
Carmel Shemmesh-Rafalowsky contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fibrous Dysplasia and McCune Albright Syndrome, run by Tovah Burstein 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 3 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 12 Aug 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Carmel Shemmesh-Rafalowsky (PI.Registry@fibrousdysplasia.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment satisfaction (time frame: Through study completion, an average of every 2 years). The study lists 6 primary outcomes in all. 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 12 Aug 2025.