Validation of the GIDS and Description of Phosphate Disorders
Organ Dysfunction ScoresHypophosphatemiaThis study will address two specific research questions simultaneously: validation of the GastroIntestinal (GI) Dysfunction score (GIDS). description of epidemiology, risk factors, and management of phosphate disorders. The aim is to recruit 20 ICUs and 1500 ICU patients. Sites will recruit all consecutive adult patients to a maximum of 120 patients or a maximum recruitment period of 8 weeks, whichever comes first. Daily data collection on gastrointestinal signs and symptoms as well as phosphate values and management will be collected during ICU stay for maximum of 7 days. 28 and 90 day mortality and days free of organ support will be the main outcomes. Secondary outcomes include prevalence of hypo- and hyperphosphatemia and description of their management.
Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic...
HypophosphatemiaHypophosphatemic Rickets2 moreA 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.
Ferric Carboxymaltose With or Without Phosphate Substitution for the Treatment of Iron Deficiency...
AnemiaIron-deficiency1 moreThis is a confirmatory trial to establish superior serum phosphate stability associated with use of Phoscap® compared with placebo as a supplement for treatment of iron deficiency or iron deficiency anemia with Ferinject® before elective surgery.
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
X-linked HypophosphatemiaThis is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab (UX023-CL303 / UX023-CL304).
Incidence of Hypophosphatemia After Treatment With Iron Isomaltoside/Ferric Derisomaltose or Ferric...
Iron Deficiency AnaemiaIron Deficiency AnemiaThe trial was designed to evaluate the incidence of unintended hypophosphatemia (low level of phosphate in the blood) in subjects with iron deficiency anaemia (IDA).
Anti-FGF23 (Burosumab) in Adult Patients With XLH
X-linked HypophosphatemiaX-linked hypophosphatemia (XLH) rare genetic disorder due by inactivating mutations in the PHEX gene leading to increased levels in FGF-23. Elevated FGF-23 reduces renal phosphate reabsorbtion and and limits 1-alpha hydroxylase driven Vitamin D activation, eventually leading to phosphate wasting, defective bone mineralization and additional health issues. Burosumab is a recombinant fully human IgG1 monoclonal antibody developed to treat XLH by binding FGF23, thereby restoring normal phosphate homeostasis. BUR03 is a Phase 3b open-label, single-arm, single-center study to confirm the efficacy and safety of Burosumab treatment in adult (age ≥18 years) XLH patients without upper age limit and irrespective of baseline pain level and to further evaluate the efficacy in this cohort and the assocaited effect of treatment on physical functioning, mobility and activity. The study aims at enrolling and treating 34 subjects with a confirmed diagnosis of XLH with q4w s.c. injection of Burosumab 1mg/kg body weight over 48 weeks. Primary objective is to attiain normal serum phosphorus levels, secondary objectives include key parameters of physical function and activity, mobility and mineral homeostasis.
A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
X-linked HypophosphatemiaThe primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.
Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic...
HypophosphatemiaFamilial1 moreThe purpose of this study is to determine the effectiveness of paricalcitol, a form of synthetic vitamin D, in lowering parathyroid hormone (PTH) levels and reducing disease symptoms in children and adults with X-linked hypophosphatemic (XLH) rickets.
Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
X-linked HypophosphatemiaThe primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
Calcitonin for Treating X-linked Hypophosphatemia
Hypophosphatemic RicketsX Linked DominantX-linked hypophosphatemia (XLH) is the most common form of inherited rickets in the United States. It also causes bone disease in adults. XLH is caused by overproduction of a hormone call FGF23, which makes the body waste phosphate. This study is designed to determine if nasal calcitonin, an already approved drug in the US, can lower blood levels of FGF23 and reduce phosphate wasting in patients with XLH. In this study the investigators will: Determine whether nasal calcitonin significantly lowers integrated 24-hour blood levels of FGF23 in patients with XLH. Evaluate whether nasal calcitonin improves serum phosphate levels in XLH. Assess whether nasal calcitonin improves blood levels of the active form of vitamin D and calcium absorption from the intestine. Make sure that nasal calcitonin is safe and well tolerated.