Natural History and Tissue Study of Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome

What this Trial Is About

Polyostotic fibrous dysplasia PFD is a rare disorder affecting multiple bones, where normal bone is replaced by abnormal fibrous tissue or weak bone. It can occur alone or with McCune-Albright Syndrome MAS, which also includes skin pigmentation and early puberty. The bone lesions can cause pain, deformity, fractures, and nerve compression, but the diseases natural history and effective treatments are not well understood. This observational study aims to define the natural history of PFD and MAS by following patients over time and collecting tissue samples for laboratory research. The study involves data collection and specimen acquisition to understand the biology of the disease, how lesions develop and change, and the related endocrine problems. Eligible subjects may also be referred to other active research studies related to PFDMAS. Participants will be followed clinically with evaluations and tissue collection as available. Researchers will analyze the data and samples to study bone biology, lesion behavior, and disease progression. The primary outcome is to successfully enroll patients and gather clinical and biological data over time. The study will continue monitoring patients to improve understanding and support future research on PFD and MAS.

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