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NCT ID: NCT06248632 Not yet recruiting - Clinical trials for X-Linked Hypophosphatemic Rickets

Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLH

FLAM-XLH
Start date: April 1, 2024
Phase:
Study type: Observational

X-linked hypophosphataemia (XLH) is a rare genetic disorder associated with increased circulating levels of the hormone FGF23, most commonly through mutation of the PHEX gene. XLH is associated with a wide range of clinical manifestations in children and adults, all of which can impact on their health-related quality of life. Conventional treatment (or standard of care, SOC) consists of phosphate supplementation and active vitamin D analogues. The management of patients with XLH has been modified in France since 2018 with the authorisation of the anti-FGF23 antibody, burosumab, in paediatrics (and in 2020 in adults). A propensity for overweight/obesity has recently been demonstrated in these patients. Could extra-skeletal effects of FGF23, in particular on the inflammatory profile of patients, be responsible for these manifestations? Obesity has been associated with inflammation in other populations. In terms of inflammation, there is a close link between FGF23 and inflammation: inflammatory cytokines increase the production of FGF23, which in turn increases inflammation by stimulating the production of inflammatory cytokines. Osteoclastogenesis and inflammation are linked and inflammation has been shown to increase bone resorption. In a recent study, the investigators showed that osteoclastogenesis was significantly impaired in cells obtained from XLH patients compared with control patients, and that osteoclasts obtained from XLH children showed higher gene expression of inflammatory markers than controls. Interestingly, no difference was observed in circulating monocytic cells between the two patient subgroups, conservative treatment and burosumab, whereas the inflammatory profile at the end of osteoclastic differentiation was reduced in cells derived from patients receiving burosumab. The aim of this study is therefore to investigate the inflammatory profile of circulating monocytic cells on the day of burosumab injection (D0) and seven days later (peak effect of anti-FGF23).

NCT ID: NCT06202027 Recruiting - Clinical trials for FGF23-related Hypophosphataemic Rickets and Osteomalacia

Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients

Start date: August 11, 2023
Phase:
Study type: Observational [Patient Registry]

The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings

NCT ID: NCT06138249 Recruiting - Hypovitaminosis D Clinical Trials

Cholecalciferol and Calcifediol Are Both Useful to Improve Vitamin D Serum Levels

VITD
Start date: July 1, 2023
Phase: N/A
Study type: Interventional

Patients with hypovitaminosis D are randomized into three arms of treatment: Group A: Calcifediol 0,266mg each month Group B: Cholecalciferol 25000UI each 15 days Group C: Calcifediol 4 drops per day. Serum levels of vitamin D are dosed after one month of treatment

NCT ID: NCT06115356 Completed - Obesity Clinical Trials

Hypovitaminosis D and "Metabolic" Inflammatory Status in Patients With Obesity

ViDO
Start date: January 1, 2018
Phase:
Study type: Observational

Since obesity is related to systemic chronic inflammatory status and hypovitaminosis D, the study aimed to assess the incidence of hypovitaminosis D in obese patients and the correlations between vitamin D levels, inflammation indices, and bioimpedance measures. A retrospective study was conducted on a cohort of obese patients. The inflammation-based prognostic scores, diagnosis of liver fibrosis, systemic inflammatory indices, and bioimpedance measures were analyzed. The linear relationship between vitamin D levels and continuous variables was assessed through the Spearman correlation coefficient, and to determine significant predictors of vitamin D levels a stepwise multiple linear regression was used.

NCT ID: NCT06065852 Recruiting - Fabry Disease Clinical Trials

National Registry of Rare Kidney Diseases

RaDaR
Start date: November 6, 2009
Phase:
Study type: Observational [Patient Registry]

The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research. The purpose of this research is to: - Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition. - Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better. - Further the development of future treatments. Participants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.

NCT ID: NCT06046820 Recruiting - Clinical trials for Generalized Arterial Calcification of Infancy

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Start date: November 5, 2023
Phase: Phase 3
Study type: Interventional

The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.

NCT ID: NCT05957692 Completed - Hypovitaminosis D Clinical Trials

Vitamin D and Adipose Tissue: a Complex Relationship

ADIPOVITAD
Start date: January 1, 2020
Phase:
Study type: Observational

The goal of this observational study is to verify the role of adipose tissue in determining the vitamin D serum level after monthly oral administration in subjects with vitamin D deficiency. The main questions it aims to answer are: - Adipose tissue represents a storage environment for vitamin D or it's an environment where vitamin D is sequestered and no longer released - On the other hands, it's possible to verify whether the adipose tissue carries out a bi-modal activity towards vitamin D - If adipose tissue exerts a bi-modal effect, it is possible to identify a specific threshold between the two effects Participants will undergo anthropometric measurements (height, weight, waist/hip ratio waist circumference) at baseline and after 6 months of intake of cholecalciferol 50,000 IU/month

NCT ID: NCT05734196 Recruiting - Clinical trials for Generalized Arterial Calcification of Infancy

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency

ENERGY
Start date: May 25, 2023
Phase: Phase 1
Study type: Interventional

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency.

NCT ID: NCT05425914 Completed - Dry Eye Syndromes Clinical Trials

Impact of Vitamin D3 Supplementation in Non-Sjogren Dry Eye Patients With Low Serum Vitamin D Level

Start date: November 1, 2021
Phase: Phase 3
Study type: Interventional

Dry eye disease is multifactorial, ocular inflammatory condition causing irritation, stinging sensation, uneasiness and blurring. Non-Sjogren syndrome occurs due to absent or dysfunction of lacrimal gland. Fat soluble vitamin D act as an agent against inflammation and its deficiency may result in various inflammatory diseases including dry eye. Purpose of this study is evaluation of vitamin D3 supplementation role in treating non-Sjogren dry eye along with conventional treatment by using artificial tears in patients with hypovitaminosis D. A prospective study was conducted in Rural health center(RHC) Buchal Kalan on 108 patients presenting with non-Sjogren dry eyes and low serum vitamin D levels. Patients were subjected to the following examination; best corrected visual acuity (BCVA), slit-lamp examination, applanation tonometry, fundoscopy, tear breakup time (TBUT) after fluorescein staining, Schirmer tear test, numerical pain rating scale (NPRS) and ocular surface disease index (OSDI) score on day 0, 15, 30, 60 and 90. Vitamin D levels was assessed by electrochemiluminescence immunoassay (ECLIA) based analyzer. The sample was randomly divided into two groups by non-probability purposive sampling. Group 1 received only artificial tears 4times/day while group 2 were given oral vitamin D3 supplementation of 6000 international unit (IU) daily along with artificial tears. Impact of oral vitamin D3 supplementation on non-Sjogren dry eyes was assessed by comparing means of ocular parameters of both groups over different period of time by using Mann-Whitney Test and Friedman Test.

NCT ID: NCT05376865 Completed - Overweight Clinical Trials

Effects of Vitamin D Supplementation on Obesity, Inflammatory and Metabolic Markers

Start date: September 29, 2021
Phase: N/A
Study type: Interventional

46 eligible overweight women with hypovitaminosis D are assigned to either vitamin D or control group using block randomization method. Vitamin D3 pearl (Zahravi Pharma Co, Tabriz, Iran) containing 50000 IU cholecalciferol or placebo are administered weekly for 12 weeks. Participants in both groups are structured to hold their sun exposure and physical activity habits and not take any vitamin supplements during the study.