Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts (HYPO-CLASTE)
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|ClinicalTrials.gov Identifier: NCT04184661|
Recruitment Status : Recruiting
First Posted : December 3, 2019
Last Update Posted : January 22, 2021
Fibroblast growth factor 23 (FGF23) is 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 Parathyroid hormone (PTH) in most tissues.
The specific role of FGF23 on bone has yet to be demonstrated. 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 (Dentin matrix acidic phosphoprotein 1 (DMP1) and Phosphate-regulating neutral endopeptidase, X-linked (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 osteoclastic biology of patients with HR compared to control patients, and to evaluate the direct impact of the treatments used in this pathology on human osteoclasts.
|Condition or disease||Intervention/treatment|
|Hypophosphatemic Rickets||Other: blood sample|
|Study Type :||Observational|
|Estimated Enrollment :||15 participants|
|Official Title:||Effect of Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts From Patients With Hypophosphatemic Rickets (HR)|
|Actual Study Start Date :||January 20, 2021|
|Estimated Primary Completion Date :||January 20, 2023|
|Estimated Study Completion Date :||January 20, 2023|
hypophosphatemic rickets patients
15 hypophosphatemic rickets patients older than 2 years will be included in this study
Other: blood sample
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.
- number of osteoclastic cells obtained after at the end of differentiation [ Time Frame: 1 day ]The analysis of osteoclastic differentiation will be obtained from the bone cells from patients with burosumab and/or 1-25 (OH) vitamin D
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04184661
|Contact: Justine BACCHETTA, PU,PH||04 27 85 61 30 ext +email@example.com|
|Contact: Sacha FLAMMIER, project Manager||0472681349 ext +firstname.lastname@example.org|
|Hôpital Femme mère enfant||Recruiting|
|Bron, France, 69677|
|Contact: Justine BACCHETTA, PU,PH 04 27 85 61 30 ext +33 email@example.com|
|Contact: Sacha FLAMMIER, project Manager 0472681349 ext +33 firstname.lastname@example.org|
|Principal Investigator: Justine BACCHETTA, PU,PH|