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Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome

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ClinicalTrials.gov Identifier: NCT00001727
Recruitment Status : Recruiting
First Posted : November 4, 1999
Last Update Posted : September 5, 2018
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Dental and Craniofacial Research (NIDCR) )

Brief Summary:

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 or disease
McCune Albright Syndrome

Detailed Description:

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.


Study Type : Observational
Estimated Enrollment : 500 participants
Official Title: Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
Study Start Date : August 25, 1998






Information from the National Library of Medicine

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Ages Eligible for Study:   up to 105 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria
  • 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
  • Patient or guardian of minor unable to provide informed consent.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00001727


Contacts
Contact: Lori C Guthrie, R.N. (301) 594-0844 guthriel@mail.nih.gov
Contact: Alison M Boyce, M.D. (301) 443-2700 alison.boyce@nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010    prpl@cc.nih.gov   
Sponsors and Collaborators
National Institute of Dental and Craniofacial Research (NIDCR)
Investigators
Principal Investigator: Alison M Boyce, M.D. National Institute of Dental and Craniofacial Research (NIDCR)

Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: National Institute of Dental and Craniofacial Research (NIDCR)
ClinicalTrials.gov Identifier: NCT00001727     History of Changes
Other Study ID Numbers: 980145
98-D-0145
First Posted: November 4, 1999    Key Record Dates
Last Update Posted: September 5, 2018
Last Verified: July 12, 2018

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Dental and Craniofacial Research (NIDCR) ):
Fibrous Dysplasia (FD)
McCune-Albright Syndrome
Polyostotic Fibrous Dysplasia

Additional relevant MeSH terms:
Syndrome
Fibrous Dysplasia, Polyostotic
Fibrous Dysplasia of Bone
Disease
Pathologic Processes
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases