Actively Recruiting
Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
Led by Hospices Civils de Lyon · Updated on 2026-04-16
20
Participants Needed
1
Research Sites
447 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
FGF23 is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a phosphaturizing agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of PTH in most tissues. The specific role of FGF23 on bone has yet to be demonstrated. In osteoblasts, overexpression of FGF23 in vitro suppresses not only osteoblastic differentiation but also the synthesis of the mineralized matrix independently of its systemic action on phosphate metabolism. In osteoblasts, FGF23 also regulates the secretion of osteopontin by directly suppressing transcription of alkaline phosphatase. In some diseases such as hypophosphatemic rickets (HR), the direct role of FGF23 on bone has not yet been studied to our knowledge, whereas these genetic hypophosphatemias are secondary to overexpression of FGF23, whether an activating mutation of FGF23 or inhibitory mutations of its inhibitors (DMP1 and PHEX). However, patients with X-linked hypophosphatemic rickets (XLH) have higher circulating FGF23 levels than healthy controls and these levels are higher in treated patients. Management of XLH consists primarily of correcting the native vitamin D defect by prescribing active vitamin D analogs as well as phosphate supplementation to improve bone mineralization and decrease dental complications, growth, and bone deformities. Recently, a new therapeutic option has been developed for XLH, burosumab, a human monoclonal antibody that binds and inhibits FGF23 activity. The use of burosumab is currently authorized in France in some pediatric patients with severe forms of XLH. Independently of the indirect bone effects of phosphate correction and vitamin D levels, the direct role of burosumab on bone cells has never been studied. The objective of this project is to study the osteoblastic biology of patients with RH compared to control patients, and to evaluate the direct impact of the treatments used in this pathology on human osteoblasts.
CONDITIONS
Official Title
Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Children from 4 months-old to 18 years-old
- Patients requiring craniosynostosis surgery followed by reference centers for rare diseases of calcium and phosphate metabolism or craniofacial malformations
- Patients and parent or holder of parental authority who have been informed of the study and do not object to participate
You will not qualify if you...
- Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery
AI-Screening
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Trial Site Locations
Total: 1 location
1
Centre de référence des craniosténoses et malformations cranio-faciales Service de neurochirurgie Pédiatrique
Bron, France, 69500
Actively Recruiting
Research Team
F
Federico DI ROCCO, MD
CONTACT
J
Justine BACCHETTA, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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