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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04184661
Other study ID # 69HCL19_0725
Secondary ID 2019-A02914-53
Status Completed
Phase
First received
Last updated
Start date January 20, 2021
Est. completion date October 27, 2021

Study information

Verified date October 2022
Source Hospices Civils de Lyon
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Completed
Enrollment 52
Est. completion date October 27, 2021
Est. primary completion date October 27, 2021
Accepts healthy volunteers No
Gender All
Age group 2 Years and older
Eligibility hypophosphatemic rickets patients: Inclusion Criteria: - children from 2 yars-old to 18 years old and adults - 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 Exclusion Criteria: - 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 Controls patients: Inclusion Criteria: - 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: - 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

Study Design


Related Conditions & MeSH terms


Intervention

Other:
blood sample
25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.

Locations

Country Name City State
France Hôpital Femme mère enfant Bron
France Hôpital Edouard Herriot Lyon
France Hôpital Bicêtre Paris Saclay Paris

Sponsors (1)

Lead Sponsor Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary number of osteoclastic cells obtained after at the end of differentiation The analysis of osteoclastic differentiation will be obtained from the bone cells from patients with burosumab and/or 1-25 (OH) vitamin D 1 day
See also
  Status Clinical Trial Phase
Recruiting NCT03748966 - Calcitriol Monotherapy for X-Linked Hypophosphatemia Early Phase 1
Active, not recruiting NCT03651505 - X-linked Hypophosphatemia Disease Monitoring Program
Completed NCT03581591 - Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets Phase 3
Active, not recruiting NCT00473187 - Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets Phase 1
Completed NCT03348644 - Milk Products in the Treatment of Hypophosphatemic Rickets N/A