2018
A Randomized, Double‐Blind, Placebo‐Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti‐FGF23 Antibody, in Adults With X‐Linked Hypophosphatemia: Week 24 Primary Analysis
Insogna KL, Briot K, Imel EA, Kamenický P, Ruppe MD, Portale AA, Weber T, Pitukcheewanont P, Cheong HI, de Beur S, Imanishi Y, Ito N, Lachmann RH, Tanaka H, Perwad F, Zhang L, Chen C, Theodore‐Oklota C, Mealiffe M, San Martin J, Carpenter TO, Investigators O. A Randomized, Double‐Blind, Placebo‐Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti‐FGF23 Antibody, in Adults With X‐Linked Hypophosphatemia: Week 24 Primary Analysis. Journal Of Bone And Mineral Research 2018, 33: 1383-1393. PMID: 29947083, DOI: 10.1002/jbmr.3475.Peer-Reviewed Original ResearchConceptsTreatment-related serious adverse eventsMean serum phosphate concentrationWOMAC physical function subscaleFibroblast growth factor 23Intact parathyroid hormoneSerious adverse eventsPhase 3 trialPhysical function subscaleChronic musculoskeletal painGrowth factor 23Serum phosphate concentrationRenal phosphate wastingHuman monoclonal antibodyLower limb deformitiesImportant medical needStiffness subscalePlacebo groupUrine calciumWorst painAdverse eventsWeek 24Dental abscessMusculoskeletal painFactor 23Function subscale
2016
Effect of KRN23, a fully human anti-FGF23 monoclonal antibody, on rickets in children with X-linked hypophosphatemia (XLH): 40-week interim results from a randomized, open-label phase 2 study
Linglart A, Carpenter T, Imel E, Boot A, Högler W, Padidela R, Hoff W, Whyte M, Chen C, Skrinar A, Agarwal S, San Martin J, Portale A. Effect of KRN23, a fully human anti-FGF23 monoclonal antibody, on rickets in children with X-linked hypophosphatemia (XLH): 40-week interim results from a randomized, open-label phase 2 study. Annales D Endocrinologie 2016, 77: 440. DOI: 10.1016/j.ando.2016.07.547.Peer-Reviewed Original ResearchRickets Severity ScoreAdverse eventsQ2W dosingXLH childrenSerum PiMost treatment-related adverse eventsOpen-label phase 2 studyTransient injection site reactionsTreatment-related adverse eventsPhase 2 studySerious adverse eventsInjection site reactionsSerum phosphate levelsRadiographic Global ImpressionSerum Pi concentrationSerum iPTHUrine calciumRenal ultrasoundSite reactionsCare treatmentGlobal ImpressionSeverity scoreKRN23Phosphorus homeostasisShort stature