Vistogard Studies 1 and 2
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.
Overview
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.
- 96 out of 100 people reached this endpoint with Uridine triacetate.
- 96 out of 100 people reached this endpoint with Uridine triacetate.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- These results apply to the people the trial enrolled: 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. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
142 analysed.
137 of 142 overdose patients survived; historical supportive-care cohort: 21 of 25 (84%) died
Source130 of 135; overdose 114 of 117 (97%), early-onset toxicity 16 of 18 (89%)
SourceSimilar pages
not linked directly; found by shared links- CompanySERB Pharmaceuticals
Shares Uridine triacetate, Emergency use of uridine triacetate for the prevention and treatment of life-threatening 5-fluorouracil and capecitabine toxicity.
- TrialA Study to Evaluate Efficacy and Safety of Tislelizumab Plus Chemotherapy for Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Adenocarcinoma and Esophageal Squamous Cell Carcinoma in Racial and Ethnic Minority Patients in the United States
Shares Fluorouracil (5-FU), Capecitabine.
- TrialA Study of HDM2017 Combination Therapy in Advanced Colorectal Cancer
Shares Fluorouracil (5-FU), Capecitabine.
- TreatmentDoxifluridine
Shares Fluorouracil (5-FU), Capecitabine.
- TrialA Study of Atezolizumab With or Without Tiragolumab Following Neoadjuvant Chemoradiotherapy in Participants With Locally Advanced Rectal Cancer
Shares Fluorouracil (5-FU), Capecitabine.
- TrialSubstudy 06C: A Study of Investigational Agents With Pembrolizumab (MK-3475) and Chemotherapy in Participants With First-Line Locally Advanced Unresectable/Metastatic Gastroesophageal Adenocarcinoma (MK-3475-06C/KEYMAKER-U06)
Shares Fluorouracil (5-FU), Capecitabine.
- TrialA Study of Zolbetuximab Together With Pembrolizumab and Chemotherapy in Adults With Gastric Cancer
Shares Fluorouracil (5-FU), Capecitabine.
- TrialTucatinib Plus Trastuzumab and Oxaliplatin-based Chemotherapy or Pembrolizumab-containing Combinations for HER2+ Gastrointestinal Cancers
Shares Fluorouracil (5-FU), Capecitabine.