2022
OR13-1 Long-Term Burosumab Therapy Provides Sustained Benefit in Patients with Tumor-Induced Osteomalacia: End of Study Findings From the Pivotal Phase 2 Study
Carpenter T, Cimms T, Hetzer J, Insogna K, Kumar R, Merritt J, Miller P, Peacock M, Rauch F, Stanciu I, Weber T, De Beur S. OR13-1 Long-Term Burosumab Therapy Provides Sustained Benefit in Patients with Tumor-Induced Osteomalacia: End of Study Findings From the Pivotal Phase 2 Study. Journal Of The Endocrine Society 2022, 6: a191-a191. PMCID: PMC9624705, DOI: 10.1210/jendso/bvac150.394.Peer-Reviewed Original ResearchTumor-induced osteomalaciaWeek 240Week 144Week 24Serum phosphorusBurosumab therapyWeek 48Bone biomarkersSafety profileNormal rangeExcess fibroblast growth factor 23SF-36 bodily pain scoresTreatment of TIOPivotal phase 2 studySF-36 physical healthSF-36 vitality scoreFibroblast growth factor 23Surface/bone surfaceBodily pain scoresMean serum phosphorusObserved safety profileRadionucleotide bone scansRare paraneoplastic syndromeBrief Pain InventoryOsteoid surface/bone surface
2021
Serum Levels of Lipocalin Are Lower in Adolescents With X-Linked Hypophosphatemia
Simpson C, Santoro A, Carpenter T, Insogna K. Serum Levels of Lipocalin Are Lower in Adolescents With X-Linked Hypophosphatemia. Journal Of The Endocrine Society 2021, 5: a27-a27. PMCID: PMC8089365, DOI: 10.1210/jendso/bvab048.052.Peer-Reviewed Original ResearchWeight/height z-scoreHeight z-scoreExcessive weight gainZ-scoreFibroblast growth factor 23Greater riskWeight gainRegular aerobic exerciseDirect pathogenic roleGrowth factor 23Impaired glucose metabolismVitamin D testingElderly normal individualsAbility of patientsAccelerated osteoarthritisAdult patientsPediatric patientsSerum levelsFactor 23Metabolic abnormalitiesAbnormal biomechanicsInsulin levelsAerobic exerciseInsulin sensitivityPathogenic role
2020
Burosumab for the Treatment of Tumor‐Induced Osteomalacia
de Beur S, Miller PD, Weber TJ, Peacock M, Insogna K, Kumar R, Rauch F, Luca D, Cimms T, Roberts MS, San Martin J, Carpenter TO. Burosumab for the Treatment of Tumor‐Induced Osteomalacia. Journal Of Bone And Mineral Research 2020, 36: 627-635. PMID: 33338281, PMCID: PMC8247961, DOI: 10.1002/jbmr.4233.Peer-Reviewed Original ResearchConceptsTumor-induced osteomalaciaCutaneous skeletal hypophosphatemia syndromeSerious adverse eventsAdverse eventsWeek 144Week 48Serum phosphorusTreatment-related adverse eventsFibroblast growth factor 23Surface/bone surfaceAcceptable safety profileOsteoid surface/bone surfacePhase 2 studyGrowth factor 23Phosphaturic mesenchymal tumorTransiliac bone biopsiesHuman monoclonal antibodyMineralization lag timePhosphate metabolismQuality of lifeDose titrationFactor 23Safety profileMesenchymal tumorsSkeletal health
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 four monthly doses of a human monoclonal anti-FGF23 antibody (KRN23) on quality of life in X-linked hypophosphatemia
Ruppe MD, Zhang X, Imel EA, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Effect of four monthly doses of a human monoclonal anti-FGF23 antibody (KRN23) on quality of life in X-linked hypophosphatemia. Bone Reports 2016, 5: 158-162. PMID: 28326356, PMCID: PMC4926842, DOI: 10.1016/j.bonr.2016.05.004.Peer-Reviewed Original ResearchSF-36v2FGF23 antibodyPatients' perceptionsPhysical functioningSF-36v2 Health SurveyFibroblast growth factor 23Physical healthMcMaster Osteoarthritis IndexDose-escalation trialAssessment of HRQoLHealth-related qualityGrowth factor 23Decreased renal tubular reabsorptionRenal tubular reabsorptionLower extremity deformitiesPoor physical healthMean scoreQuality of lifeChronic functional impairmentOsteoarthritis IndexGeneral HRQOLJoint painSecondary outcomesPhase 1/2Stiffness scores
2015
Pharmacokinetics and pharmacodynamics of a human monoclonal anti‐FGF23 antibody (KRN23) in the first multiple ascending‐dose trial treating adults with X‐linked hypophosphatemia
Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M. Pharmacokinetics and pharmacodynamics of a human monoclonal anti‐FGF23 antibody (KRN23) in the first multiple ascending‐dose trial treating adults with X‐linked hypophosphatemia. The Journal Of Clinical Pharmacology 2015, 56: 176-185. PMID: 26073451, PMCID: PMC5042055, DOI: 10.1002/jcph.570.Peer-Reviewed Original ResearchConceptsConcentration-time curveSerum fibroblast growth factor 23Multiple ascending dose trialFibroblast growth factor 23Ascending dose trialHuman IgG1 monoclonal antibodyLow-normal serumRecombinant human IgG1 monoclonal antibodyDihydroxyvitamin D concentrationsGlomerular filtration rateGrowth factor 23Serum inorganic phosphorusSerum Pi concentrationIgG1 monoclonal antibodyFGF23 antibodyDose adjustmentFactor 23Serum PiFiltration ratePhosphate reabsorptionBone markersKRN23D concentrationsNormal serumMonoclonal antibodiesProlonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23
Imel EA, Zhang X, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23. The Journal Of Clinical Endocrinology & Metabolism 2015, 100: 2565-2573. PMID: 25919461, PMCID: PMC4495171, DOI: 10.1210/jc.2015-1551.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedDose-Response Relationship, DrugDrug Administration ScheduleFamilial Hypophosphatemic RicketsFemaleFibroblast Growth Factor-23Fibroblast Growth FactorsGlomerular Filtration RateHumansImmunoglobulin GMaleMiddle AgedPhosphorusRecombinant ProteinsTreatment OutcomeYoung AdultConceptsTmP/GFRSerum PiNormal rangeOpen-label phase 1/2 studyElevated fibroblast growth factor 23Fibroblast growth factor 23Phase 1/2 studyDose-escalation studyGlomerular filtration ratePre-dose levelsGrowth factor 23Favorable safety profileMain outcome measuresProportion of subjectsAcademic medical centerPeak PiSerum inorganic phosphorusPg/mLUrinary calciumDose escalationFactor 23Monthly dosesSerum phosphorusDihydroxyvitamin DSafety profile
2011
Calcitonin Administration in X-Linked Hypophosphatemia
Liu ES, Carpenter TO, Gundberg CM, Simpson CA, Insogna KL. Calcitonin Administration in X-Linked Hypophosphatemia. New England Journal Of Medicine 2011, 364: 1678-1680. PMID: 21524226, PMCID: PMC3162370, DOI: 10.1056/nejmc1010928.Peer-Reviewed Original ResearchConceptsFibroblast growth factor 23Growth factor 23Administration of calcitoninFactor 23Calcitonin administrationHypophosphatemiaAdministrationPatientsCalcitonin