DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial

Phase 1

Recruiting

Conditions: Fibrous dysplasia/McCune-Albright syndrome
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: CTIS2024-511090-30-00

Lead Sponsor: eids Universitair Medisch Centrum (LUMC)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 82

Inclusion Criteria

Being symptomatic with an established diagnosis of FD/MAS and closed growth plates (>18 years), Pain in the region of an Fibrous dysplasia localization, not responding to adequate pain treatment and without mechanical component e.g. impending fracture, Pain score from Fibrous dysplasia 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 Na18F-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

Active pregnancy wish, pregnancy or nursing, Pain not related to Fibrous dysplasia, 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 Denosumab, 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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effect of Denosumab on pain, assessed by the difference in maximum pain scores after 6 months (2 injections);Secondary Objective: Evaluation of Denosumab effect on Quality of life, physical activity, Evaluation of possible neuropathic component of the reported pain, Evaluation of analgesics use, Evaluation of changes in mobility, Evaluation of Denosumab effect on Fibrous dysplasia lesion size and activity, Evaluation of Denosumab effect on bone density;Primary end point(s): The effect of Denosumab on pain, assessed by the difference in maximum pain score after 6 months (2 injections) by Brief Pain Inventory

Secondary Outcome Measures