Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
52 participants
OBSERVATIONAL
2021-01-20
2021-10-27
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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CASE_CONTROL
PROSPECTIVE
Study Groups
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hypophosphatemic rickets patients
30 hypophosphatemic rickets patients older than 2 years will be included in this study
blood sample
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.
controls patients
10 controls patients from pediatric nephrology unit without hypophosphatemic rickets, older than 2 years will be included in this study
blood sample
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.
Interventions
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blood sample
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.
Eligibility Criteria
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Inclusion Criteria
* patients with HR followed in the center of calcium and phosphorus metabolism rare diseases in Lyon-
* Patients and parent / holder of parental authority who have been informed of the study and do not object to participate
* children from 2 years-old to 18 years old and adults
* patients with normal renal function (Schwartz glomerular filtration rate (GFR) \>90 ml/min/1.73m²)
* Patients and parent / holder of parental authority who have been informed of the study and do not object to participate
Exclusion Criteria
* Patients under tutorship or curatorship
* Pregnant and / or breastfeeding woman
* Patient deprived of liberty
Controls patients:
* Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.
* Patients under tutorship or curatorship
* Pregnant and / or breastfeeding woman
* Patient deprived of liberty
* Patient treated with immunosuppressive drugs
* Patient with inflammatory disease
2 Years
ALL
No
Sponsors
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Hospices Civils de Lyon
OTHER
Responsible Party
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Locations
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Hôpital Femme mère enfant
Bron, , France
Hôpital Edouard Herriot
Lyon, , France
Hôpital Bicêtre Paris Saclay
Paris, , France
Countries
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Other Identifiers
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2019-A02914-53
Identifier Type: OTHER
Identifier Source: secondary_id
69HCL19_0725
Identifier Type: -
Identifier Source: org_study_id
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