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Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
This study is currently recruiting participants.
Verified by National Institutes of Health Clinical Center (CC), July 2009
First Received: November 3, 1999   Last Updated: September 3, 2009   History of Changes
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

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 200
Study Start Date: August 1998
Detailed Description:

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
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 and give informed consent.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00001727

Contacts
Contact: Patient Recruitment and Public Liaison Office (800) 411-1222 prpl@mail.cc.nih.gov
Contact: TTY 1-866-411-1010

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Additional Information:
Publications:
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

Keywords provided by National Institutes of Health Clinical Center (CC):
Bone Marrow Stromal Cells
Bone Turnover
Polyostotic Fibrous Dysplasia
McCune-Albright Syndrome

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

ClinicalTrials.gov processed this record on November 22, 2009