Trial reportThe Journal of clinical endocrinology and metabolism2025
Safety and Efficacy of Moderate-dose Denosumab in Fibrous Dysplasia: Observational Results From a Phase 2 Clinical Trial.
Trial report in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03571191 (An Open Label Pilot Study of Denosumab Treatment for Fibrous Dysplasia), which is not on this map. Cited by 6 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open Label Pilot Study of Denosumab Treatment for Fibrous Dysplasia
Who cites it
6 citing papers in PubMed, 1 synthesis or guideline pooled it.
- The use of denosumab in rare bone diseases in adults: a systematic review from the ECTS Rare Bone Disease Action Group.The Journal of clinical endocrinology and metabolism · 2026Pooled it
- A phase 2 trial of burosumab for treatment of fibroblast growth factor-23-mediated hypophosphatemia in children and adults with fibrous dysplasia.Bone research · 2026Trial
- Intensive, interdisciplinary pain treatment in fibrous dysplasia/McCune-Albright syndrome.JBMR plus · 2026Article
- Fibrous dysplasia: clinical and pathologic characteristics, surgical approaches, and outcomes in a large Italian cohort.Journal of orthopaedics and traumatology : official journal of the Italian Society of Orthopaedics and Traumatology · 2026Article
- Imaging of Fibrous Dysplasia: A Comprehensive In-Depth Analysis of Monostotic, Polyostotic, Syndromic Forms, and Bone Sarcoma Development.Journal of imaging · 2026Review
- A phase 2 trial of burosumab for treatment of fibroblast growth factor-23 mediated hypophosphatemia in children and adults with fibrous dysplasia.medRxiv : the preprint server for health sciences · 2025Article
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
contextFibrous dysplasia (FD) is a rare skeletal mosaic disease associated with fractures and disability. A phase 2 trial of the RANKL inhibitor denosumab (NCT03571191) reported profound reductions in lesion activity and increased lesional mineralization after 6 months of high-dose treatment. Denosumab was well tolerated; however, discontinuation was associated with severe hypercalcemia.
objectiveInvestigate the safety and efficacy of moderate-dose denosumab (120 mg/3 months) compared to the standard high-dose regimen.
settingClinical research center. PATIENTS: Adults with FD.
interventionsEight adults received high-dose denosumab for 6 months (120 mg/month with loading doses on weeks 2 and 3) followed by an 8-month posttreatment observation. The protocol was amended to restart moderate-dose denosumab (120 mg/3 months) if clinically indicated.
main outcome measuresBone turnover markers, 18F-sodium fluoride positron emission tomography/computed tomography (18F-NaF PET/CT), lesion biopsies.
resultsIn 6 subjects who restarted moderate-dose treatment, changes in serum markers at the initial and final dose were comparable (procollagen type 1 N-terminal propeptide -82% and -91%, C-terminal telopeptide -86% and -86% for moderate- and high-dose, respectively). There was no difference in 18F-NaF PET/CT lesional activity or absolute change in avid lesion volume between moderate- and high-dose regimens. Sequential tissue histological analyses in 1 subject demonstrated progressive lesional mineralization and reduced cellularity with moderate-dose treatment. Bone turnover markers on moderate-dose treatment showed a sustained decline in 4 subjects; however, 2 severely affected subjects developed rebound between doses, with recurrent hypercalcemia in 1 subject.
conclusionModerate-dose denosumab may provide clinical benefits comparable to the high-dose regimen in adults with FD while potentially lowering associated risks. However, discrepancies in the duration of efficacy are an important potential safety concern.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.