Actively Recruiting
Denosumab for the Treatment of Fibrous Dysplasia/McCune-Albright Syndrome in Adults: A Randomized Double-Blind Placebo-Controlled Trial
Led by Natasha Appelman-Dijkstra · Updated on 2025-01-29
82
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) 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 FD/MAS. 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 Denosumab's 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.
CONDITIONS
Brief 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 a confirmed diagnosis of Fibrous Dysplasia or McCune-Albright Syndrome and closed growth plates
- Pain in the area of an FD lesion not responding to adequate pain treatment and without mechanical causes such as impending fracture
- Pain score for maximum or average pain of 4 or higher on a visual scale
- Evidence of increased lesion activity by bone turnover markers or imaging scans
- Normal calcium, parathyroid hormone, and vitamin D levels (supplementation allowed)
- Treated hypophosphatemia with levels above 0.7 on two separate tests
- Good dental health verified within the last 12 months
You will not qualify if you...
- Active pregnancy wish, pregnancy, or breastfeeding
- Pain not related to Fibrous Dysplasia
- Uncontrolled 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
- Inability to meet study requirements
- Poor or 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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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit (in-person)
Duration - Up to 12 months
Participants receive subcutaneous injections of Denosumab 120 mg or placebo at baseline and 3 months in a double-blinded fashion. At 6 months, some participants may continue open-label Denosumab injections at 6 and 9 months depending on pain score or lesion growth.
1 baseline visit, 1 visit at 3 months, 1 visit at 6 months, and 1 visit at 9 months if continuing open-label treatment
Trial Site Locations
Total: 1 location
1
Leiden University Medical Center
Leiden, Netherlands
Actively Recruiting
Research Team
N
Natasha Appelman-Dijkstra, MD, PhD
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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