Fibrous dysplasia of bone Trials in Disease

Study on Denosumab for Treating Fibrous Dysplasia/McCune-Albright Syndrome in Adults

This clinical trial is investigating whether Denosumab, a medication that affects bone cells, can help reduce pain in adults living with Fibrous Dysplasia or McCune-Albright Syndrome. The study is being conducted in the Netherlands and will follow participants for up to 12 months.

Main inclusion criteria:

• Must be adults over 18 years old with confirmed diagnosis of Fibrous Dysplasia/McCune-Albright Syndrome and closed growth plates
• Experience ongoing pain in the affected bone area that does not improve with regular pain medications and is not caused by fractures
• Pain score must be 4 or higher on a scale that measures pain intensity (Visual Analog Scale)
• Must show increased activity in the affected bone area, detected through blood tests or imaging scans
• Must have normal levels of calcium, parathyroid hormone, and vitamin D (vitamin D supplements are allowed)
• If low phosphate levels are present, they must be treated first and reach appropriate levels
• Must have good dental health with a check-up completed within the last 12 months

Main exclusion criteria:

• Pregnant or breastfeeding women
• Individuals with severe kidney problems
• People with a history of allergic reactions to the study medication
• Those who have recently taken certain medications that affect bone health
• Individuals with other serious health conditions that might interfere with the study
• People unable to follow study procedures or attend follow-up visits

Focus and goal of the trial:

The primary goal of this study is to evaluate whether Denosumab can significantly reduce pain levels in people with Fibrous Dysplasia or McCune-Albright Syndrome. The trial will also assess how the medication affects quality of life, physical activity levels, and the need for pain medications. Participants will receive two injections of the medication over six months, given under the skin. Throughout the study, researchers will use questionnaires and imaging tests to monitor changes in pain levels, bone activity, and overall disease progression. Some participants may also receive a placebo for comparison purposes. There is an optional extension phase where participants may receive additional treatment at nine and twelve months.

Investigational drug:

The medication being tested is Denosumab, which is delivered through subcutaneous injection (under the skin). Denosumab is a type of medication called a monoclonal antibody that works by blocking a protein called RANKL. This protein is involved in the formation of osteoclasts, which are cells that break down bone tissue. By inhibiting this process, Denosumab may help reduce bone pain and improve bone health in people with Fibrous Dysplasia. The trial aims to determine whether this medication is effective specifically for managing pain associated with this rare bone condition.