{"entity":{"id":"stevie","kind":"trial","name":"STEVIE (vismodegib in ordinary practice)","aka":["STEVIE","Safety Events in Vismodegib study"],"tldr":"This is the study that measured what taking the basal cell carcinoma pill is actually like, in more than a thousand ordinary patients rather than a hundred selected ones. It shrank most tumours. Almost everybody had side effects, and the average person stopped after about eight months.","summary":"ERIVANCE licensed vismodegib on 104 patients. STEVIE was built to find out what happens when the same drug is given to everyone who might need it: 1,215 evaluable adults in 36 countries, 1,119 with locally advanced and 96 with metastatic basal cell carcinoma, treated with 150 mg daily until progression or intolerance. Safety was the primary objective, which is unusual and is the point.\n\nThe efficacy held. Investigator-assessed objective response was 68.5 per cent in locally advanced disease (95 per cent confidence interval 65.7 to 71.3) and 36.9 per cent in metastatic disease (26.6 to 48.1).\n\nThe tolerability is the finding. Ninety-eight per cent of patients had at least one treatment-emergent adverse event and 289 (23.8 per cent) had a serious one. The median treatment duration was 8.6 months, with a range from zero to 44, and only 147 patients (12 per cent) were still on the drug when the analysis was done. The characteristic events are not dangerous but are relentless and affect everything a person does: muscle spasms, loss of taste, hair loss and weight loss. Exposure beyond twelve months did not make them worse or more frequent, and most of the common events that were ongoing when treatment stopped had resolved within twelve months afterwards, which matters for anyone considering a planned break.\n\nThis is why drug holidays and intermittent schedules were tested, why a topical hedgehog inhibitor was developed, and why the second line for basal cell carcinoma is a PD-1 antibody rather than another pill down the same pathway.","status":"mixed","asOf":"2026-09-25","links":[{"label":"ClinicalTrials.gov NCT01367665","url":"https://clinicaltrials.gov/study/NCT01367665"},{"label":"Primary analysis (European Journal of Cancer 2017)","url":"https://doi.org/10.1016/j.ejca.2017.08.022"},{"label":"Primary analysis on PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/29073584/"}],"tags":[],"related":[],"cancers":["basal-cell-carcinoma","locally-advanced-bcc","skin-cancer"],"sections":[],"technologies":["hedgehog-inhibitors"],"targets":["smoothened"],"drugs":["vismodegib"],"companies":["roche-genentech"],"institutions":[],"pathways":["hedgehog"],"terms":["hedgehog-inhibitor-tolerability"],"trials":["erivance","bolt","vismoneo","cemiplimab-advanced-bcc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT01367665","phase":"2","setting":"Locally advanced or metastatic basal cell carcinoma in a population representative of clinical practice rather than of a pivotal trial: oral vismodegib 150 mg daily until progression, unacceptable toxicity or withdrawal, with safety as the primary objective","sponsor":"F. Hoffmann-La Roche","result":"Investigator-assessed response 68.5 per cent in locally advanced and 36.9 per cent in metastatic basal cell carcinoma, with treatment-emergent adverse events in 98 per cent, serious events in 23.8 per cent and a median treatment duration of 8.6 months.","yearReported":2017,"enrolled":1215,"enrolledBasis":"treated","enrolledNote":"1,215 evaluable adults from 36 countries were treated and are the population of the primary analysis; the ClinicalTrials.gov record NCT01367665 gives an enrolment of 1,232.","outcomes":[{"endpoint":"Objective response rate, locally advanced basal cell carcinoma (investigator assessed)","primary":true,"unit":"%","arms":[{"name":"Vismodegib 150 mg daily","n":1119,"value":68.5,"note":"95 per cent confidence interval 65.7 to 71.3"}],"source":"https://doi.org/10.1016/j.ejca.2017.08.022"},{"endpoint":"Objective response rate, metastatic basal cell carcinoma (investigator assessed)","unit":"%","arms":[{"name":"Vismodegib 150 mg daily","n":96,"value":36.9,"note":"95 per cent confidence interval 26.6 to 48.1"}],"source":"https://doi.org/10.1016/j.ejca.2017.08.022"},{"endpoint":"Serious treatment-emergent adverse events","primary":true,"unit":"%","arms":[{"name":"Vismodegib 150 mg daily","n":1215,"value":23.8,"note":"289 of 1,215 patients; any treatment-emergent adverse event in 98 per cent"}],"source":"https://doi.org/10.1016/j.ejca.2017.08.022"},{"endpoint":"Median duration of treatment","unit":"months","arms":[{"name":"Vismodegib 150 mg daily","n":1215,"value":8.6,"note":"range 0 to 44; 147 patients (12 per cent) still on study at the analysis"}],"source":"https://doi.org/10.1016/j.ejca.2017.08.022"}],"replication":"The Italian cohort of 182 patients reported the same pattern separately, with an overall response rate of 67.1 per cent in locally advanced disease and adverse events at the same incidence as the whole population."},"route":"/trials/stevie/","neighbours":{"cancer":[{"id":"basal-cell-carcinoma","kind":"cancer","name":"Basal cell carcinoma","route":"/cancers/basal-cell-carcinoma/"},{"id":"locally-advanced-bcc","kind":"cancer","name":"Locally advanced and metastatic basal cell carcinoma","route":"/cancers/locally-advanced-bcc/"},{"id":"skin-cancer","kind":"cancer","name":"Skin cancer (all types)","route":"/cancers/skin-cancer/"}],"technology":[{"id":"hedgehog-inhibitors","kind":"technology","name":"Hedgehog pathway inhibitors","route":"/technologies/hedgehog-inhibitors/"}],"target":[{"id":"smoothened","kind":"target","name":"Smoothened (hedgehog pathway)","route":"/targets/smoothened/"}],"drug":[{"id":"vismodegib","kind":"drug","name":"Vismodegib","route":"/drugs/vismodegib/"}],"company":[{"id":"roche-genentech","kind":"company","name":"Roche / Genentech","route":"/companies/roche-genentech/"}],"pathway":[{"id":"hedgehog","kind":"pathway","name":"Hedgehog signalling","route":"/pathways/hedgehog/"}],"term":[{"id":"hedgehog-inhibitor-tolerability","kind":"term","name":"Why people stop taking hedgehog inhibitors","route":"/terms/hedgehog-inhibitor-tolerability/"}],"trial":[{"id":"bolt","kind":"trial","name":"BOLT","route":"/trials/bolt/"},{"id":"cemiplimab-advanced-bcc","kind":"trial","name":"Cemiplimab in basal cell carcinoma after a hedgehog inhibitor","route":"/trials/cemiplimab-advanced-bcc/"},{"id":"nct06050122","kind":"trial","name":"Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome","route":"/trials/nct06050122/"},{"id":"erivance","kind":"trial","name":"ERIVANCE BCC","route":"/trials/erivance/"},{"id":"patidegib-gel-gorlin-phase-2","kind":"trial","name":"Patidegib gel in Gorlin syndrome (phase 2A)","route":"/trials/patidegib-gel-gorlin-phase-2/"},{"id":"vismoneo","kind":"trial","name":"VISMONEO (vismodegib before surgery for locally advanced basal cell carcinoma)","route":"/trials/vismoneo/"}]}}