Research Project

Project Title:	HYPO-BLASTE : Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
Project Type:	Observational Study, Enrolment not yet started
Disease group(s):	Metabolic & stone disorders, Pediatric CKD & dialysis
Project Summary:	FGF23is the cornerstone of phosphate/calcium/vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of PTH in most tissues In some diseases such as hypophosphatemic rickets, the direct role of FGF23 on bone has not yet been studied, whereas these genetic hypophosphatemias are secondary to overexpression of FGF23, whether an activating mutation of FGF23 or inhibitory mutations of its inhibitors DMP 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. The use of burosumab, a human antibody that inhibits FGF23 activity, has been recently authorized in France in pediatric patients with severe XLH forms. The objective is to study the osteoclastic biology of patients with HR compared to control patients, and to evaluate the direct impact of the treatments on osteoblasts.
Lead principal investigator(s):	Federico Di Rocco, Lyon
Co-investigator(s):	Aurelia Bertholet-Thomas, Lyon Justine Bacchetta, Lyon
Project Period:	10/2020 - 10/2022
Sponsors:	Local resources
ClinicalTrials.gov:	NCT04159675

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