NCT04159675 Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
| NCT ID | NCT04159675 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Hospices Civils de Lyon |
| Condition | Craniosynostoses |
| Study Type | OBSERVATIONAL |
| Enrollment | 20 participants |
| Start Date | 2020-09-04 |
| Primary Completion | 2029-04-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 20 participants in total. It began in 2020-09-04 with a primary completion date of 2029-04-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * 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 / craniofacial malformations * 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.
Contact & Investigator
Federico DI ROCCO, MD
PRINCIPAL INVESTIGATOR
Hospices Civils de Lyon Centre de référence des craniosténoses et malformations cranio-faciales Service de neurochirurgie Pédiatrique
Frequently Asked Questions
Who can join the NCT04159675 clinical trial?
This trial is open to participants of all sexes, aged 4 Months or older, up to 18 Years, studying Craniosynostoses. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04159675 currently recruiting?
Yes, NCT04159675 is actively recruiting participants. Contact the research team at federico.dirocco@chu-lyon.fr for enrollment information.
Where is the NCT04159675 trial being conducted?
This trial is being conducted at Bron, France.
Who is sponsoring the NCT04159675 clinical trial?
NCT04159675 is sponsored by Hospices Civils de Lyon. The principal investigator is Federico DI ROCCO, MD at Hospices Civils de Lyon Centre de référence des craniosténoses et malformations cranio-faciales Service de neurochirurgie Pédiatrique. The trial plans to enroll 20 participants.