DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD): a Randomized Double-blind Placebo-controlled Trial

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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)

Locations
Other Locations
Netherlands
Leiden University Medical Center
RECRUITING
Leiden
Contact Information
Primary
Natasha Appelman-Dijkstra, MD, PhD
n.m.appelman-dijkstra@lumc.nl
+31 625301410
Time Frame
Start Date: 2023-06-13
Estimated Completion Date: 2028-12
Participants
Target number of participants: 82
Treatments
Active_comparator: Denosumab
Denosumab randomized at baseline and 3 months in a double-blinded fashion and in case of open label at 6 and 9 months
Placebo_comparator: Placebo
Placebo randomized at baseline and 3 months in a double-blinded fashion.
Sponsors
Leads: Natasha Appelman-Dijkstra

This content was sourced from clinicaltrials.gov