Fibrous Dysplasia/McCune Albright syndrome
Conditions
Interventions
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 sc
Sponsors
Leids Universitair Medisch Centrum
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Being symptomatic with an 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 criteria
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The effect of Dmab on pain, assessed by the difference in maximum pain score after 6 months (2 injections) by Brief Pain Inventory | — |
Secondary
| Measure | Time frame |
|---|---|
| • to evaluate the effect of Dmab on average pain scores after 3, 6 months of treatment and in case of open label treatment after 9 and 12 months • to evaluate the number of patients with 50% reduction of maximal pain (BPI) after 3, 6 months of treatment and in case of open label treatment after 9 and 12 months • to evaluate the effect of Dmab on quality of life, assessed with questionaries (SF-36) at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months • to evaluate the effect of Dmab on average weekly pain assessed through a pain diary with VAS score • to investigate the effect of Dmab on Physical activity assessment (Health Assessment Questionnaire - Disability Index and screenshot of pedometer of activity during the last week on smartphone) measured at baseline, 3 months and 6 months, and in case of open label treatment after 9 and 12 months • to evaluate the prevalence of possible neuropathic component of the reported pain through Pain Detect questionnaire • to investigate the number of analgesics use and dosage used at baseline, 3 months and 6 months and in case of open label treatment after 9 and 12 months • to assess the effect of Dmab on disease activity through laboratory measurements of bone markers at baseline, 3 months and 6 months, and in case of open label treatment after 9 and 12 months • to assess the effect of Dmab on lesions activity and lesions size through bone scans at baseline and after 6 months, and in the case of open label treatment after 12 months • to assess disease quantification by nuclear imaging before and after treatment (Skeletal Burden Score (SBS) • to assess bone density and the presence of vertebral fractures (Dual-energy X-ray absorptiometry (DXA) + Vertebral Fractures Assessment (VFA) at baseline and after 12 months • to assess potential side effects in the form of Atypical femoral fractures by performing and extended DXA after 12 months | — |
Countries
Netherlands
Outcome results
None listed