{"entity":{"id":"ux023t-cl201","kind":"trial","name":"UX023T-CL201","aka":["UX023T CL201","Study 6"],"tldr":"This open-label study showed that an antibody blocking the phosphate-wasting hormone FGF23 raised blood phosphate to normal and helped heal fractures in adults whose soft-bone disease was driven by a small tumour that could not be located or removed, which supported Crysvita for that cancer-care use.","summary":"UX023T-CL201 (NCT02304367; US Crysvita label Study 6) was a multicentre, open-label, phase 2 study of burosumab, a fully human monoclonal antibody that inhibits FGF23, in adults with tumour-induced osteomalacia or cutaneous skeletal hypophosphataemia syndrome. The Journal of Bone and Mineral Research 2021 report focuses on 14 patients with tumour-induced osteomalacia, excluding two diagnosed with X-linked hypophosphataemia after enrolment and one with cutaneous skeletal hypophosphataemia syndrome.\n\nKey endpoints were changes in serum phosphorus and osteomalacia on transiliac bone biopsy at week 48. Mean serum phosphorus rose from 0.52 mmol/L at baseline and was maintained after dose titration from week 22 (0.91 mmol/L) to week 144 (0.82 mmol/L, p less than 0.0001). Most histomorphometric measures of osteomalacia improved at week 48 (osteoid volume per bone volume, osteoid thickness and mineralisation lag time fell; osteoid surface per bone surface did not change). Of 249 fractures or pseudofractures present at baseline across the 14 patients, 33 percent were fully healed and 13 percent partially healed at week 144. Patients reported less pain and fatigue and better physical health. Two patients discontinued; 16 serious adverse events occurred in seven patients and there was one death, all judged unrelated to treatment; nine patients had 16 treatment-related adverse events, all mild or moderate.\n\nThe registry lists the study as a completed single-group phase 2 with 17 participants and conditions of tumour-induced osteomalacia and epidermal nevus syndrome, neither a corpus cancer, which is why the automated pass never attached it to the burosumab record.","status":"positive","asOf":"2026-09-24","links":[{"label":"ClinicalTrials.gov NCT02304367","url":"https://clinicaltrials.gov/study/NCT02304367"},{"label":"Journal of Bone and Mineral Research 2021","url":"https://doi.org/10.1002/jbmr.4233"},{"label":"Crysvita label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=102f96a0-6e3a-4fc1-b204-34d604683af6"},{"label":"Crysvita EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/crysvita"}],"tags":[],"related":[],"cancers":[],"sections":["supportive-care"],"technologies":["monoclonal-antibody"],"targets":[],"drugs":["burosumab"],"companies":["kyowa-kirin"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-ux023t-cl201-jan-de-beur-jbmr-2021"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02304367","phase":"2","setting":"Adults with tumour-induced osteomalacia from phosphaturic mesenchymal tumours that cannot be found or removed: open-label burosumab with dose titration, for FGF23-driven phosphate wasting","sponsor":"Kyowa Kirin","result":"Serum phosphorus rose from 0.52 mmol/L at baseline to 0.82 mmol/L at week 144 (p<0.0001); 33% of baseline fractures or pseudofractures fully healed and 13% partially healed by week 144.","yearReported":2021,"enrolled":14,"enrolledBasis":"analysed","enrolledNote":"The Journal of Bone and Mineral Research 2021 report analyses 14 adults with tumour-induced osteomalacia after excluding two patients found to have X-linked hypophosphataemia after enrolment and one with cutaneous skeletal hypophosphataemia syndrome; ClinicalTrials.gov NCT02304367 lists an actual enrolment of 17.","outcomes":[{"endpoint":"Mean serum phosphorus at week 144 (tumour-induced osteomalacia cohort)","primary":true,"unit":"mmol/L","arms":[{"name":"Burosumab","n":14,"value":0.82,"note":"Baseline 0.52 mmol/L; 0.91 mmol/L at week 22 after dose titration"}],"p":"<0.0001","source":"https://doi.org/10.1002/jbmr.4233"},{"endpoint":"Baseline fractures or pseudofractures fully healed at week 144","unit":"%","arms":[{"name":"Burosumab","n":14,"value":33,"note":"Of 249 fractures or pseudofractures present at baseline across 14 patients; a further 13% partially healed"}],"source":"https://doi.org/10.1002/jbmr.4233"}],"replication":"A separate 13-patient open-label study in Japan and Korea (US Crysvita label Study 7; Imanishi 2021) reported the same direction of effect and is not recorded here."},"route":"/trials/ux023t-cl201/","neighbours":{"section":[{"id":"supportive-care","kind":"section","name":"Supportive Care & Survivorship","route":"/fronts/supportive-care/"}],"technology":[{"id":"monoclonal-antibody","kind":"technology","name":"Monoclonal antibodies","route":"/technologies/monoclonal-antibody/"}],"drug":[{"id":"burosumab","kind":"drug","name":"Burosumab","route":"/drugs/burosumab/"}],"company":[{"id":"kyowa-kirin","kind":"company","name":"Kyowa Kirin","route":"/companies/kyowa-kirin/"}],"paper":[{"id":"paper-ux023t-cl201-jan-de-beur-jbmr-2021","kind":"paper","name":"Burosumab for the treatment of tumor-induced osteomalacia (UX023T-CL201)","route":"/key-papers/paper-ux023t-cl201-jan-de-beur-jbmr-2021/"}]}}