DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD) — DeFiD  

Fibrous Dysplasia Clinical Trial

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

Status Recruiting

Clinical Trial 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.

Clinical Trial 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 score <4 will exit the study to discontinue study medication and proceed in usual care, while patients with pain score ≥4 or lesional growth will be offered Dmab 120 mg at 6 and 9 months in an open-label design. ;

Related Conditions & MeSH terms

• Fibrous Dysplasia
• Fibrous Dysplasia of Bone
• McCune Albright Syndrome
• Syndrome

• NCT number: NCT05966064
• Study type: Interventional
• Source: Leiden University Medical Center
• Contact: Natasha Appelman-Dijkstra, MD, PhD
• Phone: +31 625301410
• Email: n.m.appelman-dijkstra@lumc.nl
• Status: Recruiting
• Phase: Phase 4
• Start date: June 13, 2023
• Completion date: June 2025