{"entity":{"id":"vistogard-studies-1-2","kind":"trial","name":"Vistogard Studies 1 and 2","aka":["401.10.001","Uridine triacetate expanded-access studies"],"tldr":"Vistogard Studies 1 and 2 gave an oral uridine antidote quickly after a fluorouracil or capecitabine overdose or early severe toxicity; almost everyone treated survived, while most people in an earlier group given supportive care alone had died, and the studies were the main evidence for the US approval of Vistogard.","summary":"Vistogard Studies 1 and 2 were two open-label expanded-access studies of oral uridine triacetate for adults and children after a fluorouracil or capecitabine overdose, or with early-onset severe or life-threatening toxicity. Uridine triacetate was started as soon as possible, in most patients within 96 hours of the fluorouracil or capecitabine; the label dose is 10 g by mouth every six hours for 20 doses, with a body-surface-area dose for young children. Outcomes were survival, resumption of chemotherapy and safety, and survival was compared with a historical cohort of overdose patients who received only supportive care.\n\nIn the Cancer 2017 combined analysis, 137 of 142 overdose patients (96 percent) treated with uridine triacetate survived and had rapid reversal of severe acute cardiotoxicity and neurotoxicity. In the historical supportive-care cohort, 21 of 25 patients (84 percent) died. Among 141 treated overdose patients with a cancer diagnosis, 53 resumed chemotherapy (Cancer 2017).\n\nThe US Vistogard label names the same programme as Study 1 (60 patients) and Study 2 (75 patients), pools 135 patients (117 overdose, 18 early-onset toxicity), and reports survival at 30 days or until chemotherapy resumed of 130 of 135 (96 percent), with 114 of 117 (97 percent) in the overdose group, against the same historical 25-patient supportive-care cohort in which 84 percent died. The FDA approved Vistogard in December 2015. The registry lists the Wellstat expanded-access protocol under org id 401.10.001 as approved for marketing, without a phase or enrolment count. The programme treats overdose and early severe toxicity rather than a cancer, so it carries no cancer ids.","status":"positive","asOf":"2026-09-24","links":[{"label":"ClinicalTrials.gov NCT01432301","url":"https://clinicaltrials.gov/study/NCT01432301"},{"label":"Cancer 2017","url":"https://doi.org/10.1002/cncr.30321"},{"label":"Vistogard label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=269f7363-63ed-444e-85f1-f0009e44818b"}],"tags":[],"related":["fluorouracil","capecitabine"],"cancers":[],"sections":["supportive-care"],"technologies":[],"targets":[],"drugs":["uridine-triacetate"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-vistogard-cancer-2017"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT01432301","phase":"2","setting":"Emergency treatment after fluorouracil or capecitabine overdose, or early-onset severe or life-threatening toxicity within 96 hours: oral uridine triacetate compared with a historical supportive-care overdose cohort","sponsor":"Wellstat Therapeutics","result":"Survival 96% (137 of 142) with uridine triacetate; historical supportive-care overdose cohort mortality 84% (21 of 25).","yearReported":2017,"enrolled":142,"enrolledBasis":"analysed","enrolledNote":"The Cancer 2017 abstract reports survival for 142 overdose patients, the Vistogard label pools Study 1 (n=60) and Study 2 (n=75) as 135 patients, and ClinicalTrials.gov NCT01432301 (expanded access, org id 401.10.001) posts no enrolment count.","outcomes":[{"endpoint":"Survival (overdose patients)","primary":true,"unit":"%","arms":[{"name":"Uridine triacetate","n":142,"value":96,"note":"137 of 142 overdose patients survived; historical supportive-care cohort: 21 of 25 (84%) died"}],"source":"https://doi.org/10.1002/cncr.30321"},{"endpoint":"Survival at 30 days or until chemotherapy resumed (US label, Studies 1 and 2 combined)","unit":"%","arms":[{"name":"Uridine triacetate","n":135,"value":96,"note":"130 of 135; overdose 114 of 117 (97%), early-onset toxicity 16 of 18 (89%)"}],"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=269f7363-63ed-444e-85f1-f0009e44818b"}]},"route":"/trials/vistogard-studies-1-2/","neighbours":{"drug":[{"id":"capecitabine","kind":"drug","name":"Capecitabine","route":"/drugs/capecitabine/"},{"id":"fluorouracil","kind":"drug","name":"Fluorouracil (5-FU)","route":"/drugs/fluorouracil/"},{"id":"uridine-triacetate","kind":"drug","name":"Uridine triacetate","route":"/drugs/uridine-triacetate/"}],"section":[{"id":"supportive-care","kind":"section","name":"Supportive Care & Survivorship","route":"/fronts/supportive-care/"}],"paper":[{"id":"paper-vistogard-cancer-2017","kind":"paper","name":"Emergency use of uridine triacetate for the prevention and treatment of life-threatening 5-fluorouracil and capecitabine toxicity","route":"/key-papers/paper-vistogard-cancer-2017/"}]}}