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

NCT05966064 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 82

Last updated 2025-01-29

No results posted yet for this study

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.

Conditions

  • Fibrous Dysplasia
  • McCune Albright Syndrome

Interventions

DRUG

Denosumab 120 Mg/1.7 Ml Inj

Denosumab randomized at baseline and after 3 months at 6 and 9 months in case of open label

DRUG

Placebo

placebo randomized at baseline and after 3 months

Sponsors & Collaborators

  • Natasha Appelman-Dijkstra

    lead OTHER

Principal Investigators

  • Natasha Appelman-Dijsktra, MD, PhD · Leiden University Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-13
Primary Completion
2027-12-31
Completion
2028-12-31

Countries

  • Netherlands

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05966064 on ClinicalTrials.gov