{"entity":{"id":"enliven","kind":"trial","name":"ENLIVEN","aka":[],"tldr":"The CSF1R blocker pexidartinib shrank tenosynovial giant cell tumours in about four in ten patients against none on placebo, becoming the first approved drug for this joint tumour, with a liver-safety programme attached.","summary":"ENLIVEN randomised 120 patients with symptomatic, advanced tenosynovial giant cell tumour in whom surgery would cause worsening functional limitation or severe morbidity to pexidartinib or placebo for 24 weeks. The RECIST response rate at week 25 was 39 percent with pexidartinib versus none with placebo, with improvements in range of motion and function; mixed or cholestatic liver injury in a minority of patients led to a risk evaluation and mitigation strategy at FDA approval in August 2019. Published in the Lancet in 2019, it was the first randomised trial to show benefit of a drug in this disease and set the template for vimseltinib (MOTION) and emactuzumab.","status":"positive","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT02371369","url":"https://clinicaltrials.gov/study/NCT02371369"},{"label":"Lancet 2019","url":"https://doi.org/10.1016/S0140-6736(19)30764-0"}],"tags":["subtype-page"],"related":[],"cancers":["tenosynovial-giant-cell-tumour","sarcoma"],"sections":[],"technologies":[],"targets":["csf1r"],"drugs":["pexidartinib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02371369","phase":"3","setting":"Symptomatic tenosynovial giant cell tumour not amenable to surgery: pexidartinib versus placebo","sponsor":"Daiichi Sankyo (Plexxikon)","result":"Week 25 RECIST response 39% vs 0% (placebo); FDA approval 2019 with a liver-toxicity REMS.","yearReported":2019,"enrolled":120,"outcomes":[{"endpoint":"Objective response at week 25 (RECIST)","primary":true,"unit":"percent","arms":[{"name":"Pexidartinib","n":61,"value":39},{"name":"Placebo","n":59,"value":0}],"p":"<0.0001","source":"https://doi.org/10.1016/S0140-6736(19)30764-0"}]},"route":"/trials/enliven/","neighbours":{"cancer":[{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"},{"id":"tenosynovial-giant-cell-tumour","kind":"cancer","name":"Tenosynovial giant cell tumour (TGCT)","route":"/cancers/tenosynovial-giant-cell-tumour/"}],"target":[{"id":"csf1r","kind":"target","name":"CSF1R","route":"/targets/csf1r/"}],"drug":[{"id":"pexidartinib","kind":"drug","name":"Pexidartinib","route":"/drugs/pexidartinib/"}]}}