Actively Recruiting
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Led by Natasha Appelman-Dijkstra · Updated on 2025-01-29
82
Participants Needed
1
Research Sites
285 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease, consisting of the replacement of normal bone tissue with fibrous tissue. FD lesions may be isolated in one or more bones or may be associated with endocrinopathies in McCune-Albright syndrome. Bone lesions constitute of weak bone tissue, leading to higher risk of fractures, pain and decreased quality of life. There is no cure for FD lesions and current therapies failed to soothe patients' complaints or to display any effect on progression of the lesions on imaging. However, the RANKL-inhibitor Denosumab demonstrated encouraging results in mouse models and in off-label clinical use, leading to clinical, biochemical and radiographical improvements. Study's aim is to investigate whether 3-monthly Denosumab will improve the clinical, radiological and biochemical manifestations of FD bone lesions.
CONDITIONS
Official Title
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults over 18 years with confirmed symptomatic Fibrous Dysplasia/McCune-Albright syndrome and closed growth plates
- Pain in the Fibrous Dysplasia area not responding to adequate pain treatment and without mechanical causes
- Pain score of 4 or higher on a visual scale for the affected lesion
- Increased activity in bone lesions shown by markers or imaging tests
- Normal calcium, parathyroid hormone, and vitamin D levels (supplementation allowed)
- Treated low phosphate levels confirmed by testing
- Good dental health confirmed within the last 12 months
You will not qualify if you...
- Pregnancy, nursing, or active pregnancy plans
- Pain not related to Fibrous Dysplasia
- Uncontrolled hormonal or endocrine diseases
- Untreated vitamin D deficiency, low calcium, or low phosphate levels
- Use of bisphosphonates or Denosumab within 6 months before the study
- History of severe side effects from Denosumab
- Unable to meet study requirements
- Poor untreated dental health without plans for treatment
- Use of other bone-affecting drugs like high-dose corticosteroids
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Leiden University Medical Center
Leiden, Netherlands
Actively Recruiting
Research Team
N
Natasha Appelman-Dijkstra, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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