View Clinical Trial (Medical Research Study)
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
| City: |
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Bethesda |
| State: |
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Maryland |
| Zip Code: |
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20892 |
| Conditions: |
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Polyostotic Fibrous Dysplasia |
| Purpose: |
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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.
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| Study Summary: |
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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.
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| Criteria: |
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- INCLUSION CRITERIA
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.
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| NCT ID: |
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NCT00001727 |
| Primary Contact: |
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Principal Investigator
Michael T Collins, M.D.
National Institute of Dental and Craniofacial Research (NIDCR)
Michael T Collins, M.D.
Phone: (301) 496-4563
Email: mcollins@mail.nih.gov
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| Backup Contact: |
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N/A |
| Location Contact: |
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Bethesda, Maryland 20892
United States
For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)
Phone: 800-411-1222
Email: prpl@mail.cc.nih.gov
Site Status: Recruiting |
| Data Source: |
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ClinicalTrials.gov |
| Date Processed: |
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May 24, 2013 |
| Modifications to this listing: |
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