Denosumab Treatment for Fibrous Dysplasia and McCune-Albright Syndrome in Adults A Randomized, Double-Blind, Placebo-Controlled Study

What this Trial Is About

Fibrous DysplasiaMcCune-Albright syndrome FDMAS is a rare condition where normal bone is replaced by fibrous tissue, leading to weak bones that may cause fractures, pain, and decreased quality of life. Researchers are studying whether the medication Denosumab, given every three months, can improve symptoms and slow the progression of FD bone lesions. This study is a phase 4 trial aiming to evaluate clinical, radiological, and biochemical effects of Denosumab in adults with FDMAS. Participants are randomly assigned to receive either Denosumab 120 mg or a placebo through injections under the skin at the start and after three months. After two injections, those with low pain scores stop the study treatment and continue usual care, while those with higher pain or lesion growth receive open-label Denosumab injections at six and nine months. This design allows comparison of Denosumabs effects against placebo initially and extended treatment for those needing it. Throughout the study, participants will have their pain levels, quality of life, physical activity, bone markers, lesion size and activity, bone density, and fracture occurrence monitored at multiple points up to 12 months. Weekly pain scores and medication use for pain relief will also be tracked. Safety assessments include watching for potential side effects like atypical femoral fractures. The total participation duration can last up to one year, including blinded and open-label treatment phases with regular clinical and imaging evaluations.

DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)