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Hypophosphatemic Rickets clinical trials

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NCT ID: NCT04184661 Completed - Clinical trials for Hypophosphatemic Rickets

Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts

HYPO-CLASTE
Start date: January 20, 2021
Phase:
Study type: Observational

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.

NCT ID: NCT03748966 Recruiting - Clinical trials for X-linked Hypophosphatemia

Calcitriol Monotherapy for X-Linked Hypophosphatemia

Start date: March 28, 2019
Phase: Early Phase 1
Study type: Interventional

Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose will be escalated during the first 3 months of therapy. The investigators hypothesize that treatment of adults and children with XLH alone will improve serum phosphate levels and skeletal mineralization without causing an increase in kidney calcifications. The study will also examine if calcitriol therapy will improve growth in children.

NCT ID: NCT03651505 Active, not recruiting - Clinical trials for X-linked Hypophosphatemia

X-linked Hypophosphatemia Disease Monitoring Program

Start date: July 16, 2018
Phase:
Study type: Observational

The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.

NCT ID: NCT03581591 Completed - Pain, Chronic Clinical Trials

Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

Start date: January 31, 2018
Phase: Phase 3
Study type: Interventional

A 52 week, open label trial to assess the safety and efficacy of KRN23, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome(ENS) and associated hypophosphatemic rickets A 26 weeks extension to original study to monitor patient lab results for her safety.

NCT ID: NCT03348644 Completed - Clinical trials for Hypophosphatemic Rickets

Milk Products in the Treatment of Hypophosphatemic Rickets

Start date: August 1, 2015
Phase: N/A
Study type: Interventional

The aim of this study was to investigate the feasibility and efficacy of a high intake of milk and/or cheese products compared to phosphate tablets in patients with hypophosphatemic rickets when evaluating the S-phosphate levels as a main effect parameter. The study was designed as a randomized, multiple crossover study.

NCT ID: NCT00473187 Active, not recruiting - Growth Disorders Clinical Trials

Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets

Start date: August 2004
Phase: Phase 1
Study type: Interventional

X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, impaired renal phosphate reabsorption and vitamin D metabolism. Despite oral phosphate and vitamin D treatment, most children with XLH demonstrate reduced adult height. The main objective of the study is to determine the beneficial effects of recombinant human growth hormone (rhGH) therapy on body proportions after 36 month in these patients. Secondary objective is to monitor side effects of the therapy.