RecruitingRare disease

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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Denosumab on a condition known as Fibrous Dysplasia/McCune-Albright Syndrome in adults. Fibrous Dysplasia is a rare bone disorder where normal bone is replaced with fibrous tissue, leading to pain and fractures. McCune-Albright Syndrome is a genetic disorder that affects the bones, skin, and several hormone-producing tissues. The purpose of this study is to evaluate how Denosumab affects pain levels in patients with this condition.

Participants in the study will receive Denosumab through subcutaneous injection, which means the medication is injected under the skin. The study will also include a placebo group for comparison. The trial will last for a period of up to 12 months, during which participants will receive two injections over the first six months. The study will assess the impact of Denosumab on pain by comparing pain scores before and after treatment. Additionally, the study will look at other factors such as quality of life, physical activity, and the use of pain medications.

Throughout the study, participants will undergo various assessments, including questionnaires and imaging tests, to monitor changes in their condition. The study aims to provide valuable information on the effectiveness of Denosumab in managing pain and improving the quality of life for individuals with Fibrous Dysplasia/McCune-Albright Syndrome. This research could potentially lead to better treatment options for those affected by this rare condition.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the diagnosis of fibrous dysplasia or McCune-Albright syndrome, ensuring closed growth plates, and confirming a pain score of 4 or higher on a visual scale.
Additional tests may include checking bone turnover markers and ensuring normal levels of calcium, parathyroid hormone, and vitamin D.
Step 2
Baseline measurements
Baseline measurements are taken to assess pain levels, quality of life, physical activity, and disease activity. This involves completing questionnaires and undergoing imaging tests such as bone scans and dual-energy X-ray absorptiometry (DXA).
Step 3
First denosumab injection
The first dose of denosumab is administered via subcutaneous injection. This medication is intended to help manage pain associated with fibrous dysplasia.
Step 4
Follow-up assessments at 3 months
After three months, follow-up assessments are conducted. These include evaluating pain levels, quality of life, and physical activity. Questionnaires and imaging tests are repeated to monitor changes.
Step 5
Second denosumab injection
A second dose of denosumab is administered via subcutaneous injection six months after the first dose.
Step 6
Follow-up assessments at 6 months
Six months into the study, further assessments are conducted to evaluate the effect of denosumab on pain and quality of life. Imaging tests and questionnaires are used to gather data.
Step 7
Optional open-label treatment
Participants may be offered open-label treatment, which involves additional assessments and potential further doses of denosumab at nine and twelve months. This phase includes continued monitoring of pain, quality of life, and disease activity.
Step 8
Final assessments
At the end of the study, final assessments are conducted to evaluate the overall impact of denosumab on pain and disease progression. This includes a comprehensive review of all collected data and imaging results.

Who can join the trial?

8 criteria

Must have symptoms and a confirmed diagnosis of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS) with closed growth plates, meaning you are over 18 years old.
Experience pain in the area affected by fibrous dysplasia that does not improve with regular pain treatment and is not due to a mechanical issue like a possible fracture.
Have a pain score of 4 or higher on a scale called the Visual Analog Scale (VAS), which measures pain intensity.
Show increased activity in the affected bone area, which can be identified by higher levels of certain markers in the blood or increased activity seen in specific imaging tests like Na18F-PET/CT or bone scans.
Have normal levels of calcium, parathyroid hormone, and vitamin D in the body. Taking vitamin D supplements is allowed.
If you have low phosphate levels, they must be treated and measured as above 0.7 in two separate tests.
Maintain good dental health, with a dental check-up done within the last 12 months.
Both males and females are eligible to participate.

Who cannot join the trial?

6 criteria

Patients who are pregnant or breastfeeding cannot participate.
Individuals with severe kidney problems are excluded.
People with a history of allergic reactions to the study medication are not allowed.
Participants who have received certain medications that affect bone health recently are excluded.
Individuals with other serious health conditions that might interfere with the study are not eligible.
Patients who are unable to follow the study procedures or attend follow-up visits cannot participate.

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Responsive
Leids Universitair Medisch Centrum (LUMC)
Leiden, The Netherlands

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Investigated drugs

Denosumab is a medication being studied for its potential to treat Fibrous Dysplasia/McCune-Albright Syndrome in adults. The trial aims to evaluate how effective Denosumab is in reducing pain associated with this condition. Participants in the study receive injections of Denosumab, and the main goal is to see if there is a significant decrease in their pain levels after six months of treatment.

What is already known about the treatment

Denosumab – Denosumab is administered via subcutaneous injection and is currently being studied in clinical trials for its effectiveness in treating fibrous dysplasia and McCune-Albright syndrome. It is a well-documented medication in medical literature, primarily used for conditions involving bone loss. The main therapeutic indication for Denosumab in this trial is to reduce pain associated with these conditions. At the molecular level, Denosumab works by inhibiting a protein called RANKL, which is involved in the formation and function of osteoclasts, the cells responsible for bone resorption. It is classified pharmacologically as a monoclonal antibody.

Investigated diseases

Fibrous Dysplasia/McCune-Albright Syndrome – This is a rare genetic disorder that affects the bones, skin, and endocrine system. It is characterized by the replacement of normal bone with fibrous tissue, leading to bone deformities, fractures, and pain. The condition can also cause skin pigmentation changes and hormonal imbalances, such as early puberty. The severity and symptoms can vary widely among individuals, with some experiencing mild effects and others facing significant challenges. The disease often progresses during childhood and adolescence, with bone lesions typically stabilizing in adulthood. The syndrome is caused by mutations in the GNAS gene, which occur after conception and are not inherited.