# Vistogard Studies 1 and 2

Source: https://onco.cc/trials/vistogard-studies-1-2/  
OnCo record `vistogard-studies-1-2` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

In 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).

The 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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-24
- Also known as: 401.10.001; Uridine triacetate expanded-access studies
- Registry id: 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
- Enrolled: 142
- Result: Survival 96% (137 of 142) with uridine triacetate; historical supportive-care overdose cohort mortality 84% (21 of 25).
- Outcomes: Survival (overdose patients): Uridine triacetate 96%; Survival at 30 days or until chemotherapy resumed (US label, Studies 1 and 2 combined): Uridine triacetate 96%

## Sources

- ClinicalTrials.gov NCT01432301: https://clinicaltrials.gov/study/NCT01432301
- Cancer 2017: https://doi.org/10.1002/cncr.30321
- Vistogard label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=269f7363-63ed-444e-85f1-f0009e44818b

## Connected records

- drugs: [Capecitabine](https://onco.cc/drugs/capecitabine/), [Fluorouracil (5-FU)](https://onco.cc/drugs/fluorouracil/), [Uridine triacetate](https://onco.cc/drugs/uridine-triacetate/)
- fronts: [Supportive Care & Survivorship](https://onco.cc/fronts/supportive-care/)
- key papers: [Emergency use of uridine triacetate for the prevention and treatment of life-threatening 5-fluorouracil and capecitabine toxicity](https://onco.cc/key-papers/paper-vistogard-cancer-2017/)

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