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| Sponsor: | National Institute of Dental and Craniofacial Research (NIDCR) |
|---|---|
| Information provided by: | National Institutes of Health Clinical Center (CC) |
| ClinicalTrials.gov Identifier: | NCT00001727 |
Purpose
Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful, and depending on the location of the lesion, can cause significant morbidity. Lesions in weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined systemic therapies for the bone disease. The purpose of this study is to define the natural history of the disease with or without treatment.
| Condition |
|---|
|
Polyostotic Fibrous Dysplasia |
| Study Type: | Observational |
| Official Title: | Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome |
| Estimated Enrollment: | 200 |
| Study Start Date: | August 1998 |
Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly reabsorbed and replaced by abnormal fibrous tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful, and depending on the location of the lesion, can cause significant morbidity. Lesions in weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined systemic therapies for the bone disease. The purpose of this study is to define the natural history of the disease with or without treatment.
Eligibility| Ages Eligible for Study: | 1 Year and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Any patient with a likelihood of having PFD or MAS, based on information from a referring physician or surgeon or provided by the patient or guardian, will be eligible for consideration for inclusion in the study. The diagnosis will be based on typical findings on bone biopsy, or on clinical grounds.
EXCLUSION CRITERIA
Patient, child or parents unwilling to fully cooperate with the evaluation and give informed consent.
Contacts and Locations| Contact: Patient Recruitment and Public Liaison Office | (800) 411-1222 | prpl@mail.cc.nih.gov |
| Contact: TTY | 1-866-411-1010 |
| United States, Maryland | |
| National Institutes of Health Clinical Center, 9000 Rockville Pike | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
More Information
| Study ID Numbers: | 980145, 98-D-0145 |
| Study First Received: | November 3, 1999 |
| Last Updated: | September 3, 2009 |
| ClinicalTrials.gov Identifier: | NCT00001727 History of Changes |
| Health Authority: | United States: Federal Government |
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Bone Marrow Stromal Cells Bone Turnover Polyostotic Fibrous Dysplasia McCune-Albright Syndrome |
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Pathologic Processes Disease Musculoskeletal Diseases Syndrome Bone Diseases, Developmental |
Osteochondrodysplasias Fibrous Dysplasia of Bone Fibrous Dysplasia, Polyostotic Bone Diseases |