Understanding the natural history of Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
This study is trying to learn more about how Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome affect patients over time by collecting data and samples from them.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Ages | 1 Day to 100 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT00001727 on ClinicalTrials.gov |
What this trial studies
This observational study aims to collect data and biological samples from patients diagnosed with Polyostotic Fibrous Dysplasia (PFD) and McCune-Albright Syndrome (MAS). It seeks to define the natural history of these conditions by following patients over time and conducting in vitro experiments with their samples. The study will help to better understand the progression and impact of these disorders, which are characterized by disfiguring and painful bone lesions. Currently, there are no clearly defined systemic therapies for these conditions, making this research particularly important.
Who should consider this trial
Good fit: Ideal candidates for this study include individuals of any age who are suspected to have PFD or MAS based on clinical evaluation.
Not a fit: Patients who are unwilling to cooperate with evaluations or cannot provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the natural progression of PFD and MAS, potentially leading to improved management strategies for affected patients.
How similar studies have performed: While there is limited research on the natural history of these conditions, this study represents a novel approach to understanding PFD and MAS.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA 1. Any patient, age 1 day of life and older, with a likelihood of having PFD or MAS, based on information from an appropriate referring physician or surgeon or provided by the patient or guardian. The diagnosis will be based on typical findings on bone biopsy or on clinical grounds. EXCLUSION CRITERIA 1. Patient, child, or parent/guardian unwilling to fully cooperate with the evaluations. 2. Patient or parent/guardian unable to provide informed consent.
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Alison M Boyce, M.D. — National Institute of Dental and Craniofacial Research (NIDCR)
- Study coordinator: Olivia J de Jong, C.R.N.P.
- Email: olivia.dejong@nih.gov
- Phone: (240) 595-2764
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.