Status:
RECRUITING
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Lead Sponsor:
Natasha Appelman-Dijkstra
Conditions:
Fibrous Dysplasia
McCune Albright Syndrome
Eligibility:
All Genders
18+ years
Phase:
PHASE4
Brief Summary
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 b...
Detailed Description
Eligible patients will be randomized to treatment with either subcutaneous Dmab 120mg or placebo at baseline and 3 months in a blinded fashion. At 6 months, after 2 injections, patients with pain scor...
Eligibility Criteria
Inclusion
- Symptomatic patients with established diagnosis of FD/MAS and closed growth plates(\>18 years)
- Pain in the region of an FD localization, not responding to adequate pain treatment and without mechanical component e.g. impending fracture
- Pain score from FD lesion for maximum or average pain on VAS ≥ 4
- Increased lesional activity defined as increased bone turnover markers (ALP, P1NP or CTX) or increased activity on Na\[18F\]-PET/CT or bone scintigraphy in at least one lesion
- Normal levels of calcium, parathyroid hormone and vitamin D (supplementation is allowed)
- Treated hypophosphatemia (defined as \>0.7 at two separate measures)
- good dental health (last check within the last 12 months)
Exclusion
- Active pregnancy wish, pregnancy or nursing
- Pain not related to FD
- Uncontrolled endocrine disease
- Untreated vitamin D deficiency, hypocalcemia or hypophosphatemia
- Previous use of bisphosphonates or Dmab \< 6 months before inclusion ('6 months wash out')
- Previously reported severe side effects on Dmab
- Inability to fulfil study requirements
- Poor untreated dental health without intention to get treatment
- Treatment with other bone influencing drugs, such as high doses corticosteroids
Key Trial Info
Start Date :
June 13 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
82 Patients enrolled
Trial Details
Trial ID
NCT05966064
Start Date
June 13 2023
End Date
December 1 2028
Last Update
January 29 2025
Active Locations (1)
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1
Leiden University Medical Center
Leiden, Netherlands