Dose Optimization and Efficacy Assessment of a Fluoropyrimidine Antidote (NCT07032142) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Dose Optimization and Efficacy Assessment of a Fluoropyrimidine Antidote
Brazil66 participantsStarted 2025-07
Plain-language summary
Fluoropyrimidines (FLU) are drugs widely used in chemotherapy for various tumors, such as breast, colon, rectal, and gastric cancers. FLU is a drug that inhibits thymine synthesis and, consequently, DNA synthesis, leading to tumor cell death. However, up to 30% of patients treated with FLU experience severe toxicities, depending on the dose and regimen received. The most common symptoms include mucositis, vomiting, nausea, diarrhea, and neutropenia.
The enzyme dihydropyrimidine dehydrogenase (DPD) plays a key role in FLU metabolism. Patients with mutations in the DPYD gene (which encodes DPD) are at high risk of experiencing severe toxicities from FLU. Uridine triacetate (UT) is a drug that can be used as an antidote for 5-FU in patients who develop severe toxicities. However, despite its efficacy, it is expensive and not commercially available in Brazil.
Currently, the Brazilian population has no access to an antidote for the treatment of FLU-related toxicities. This Phase I/II study will evaluate the dose, safety, and efficacy of compound the association of two molecules as an antidote for grade 3 or higher toxicities resulting from the use of FLU.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Presence of at least one severe toxicity or intoxication resulting from fluoropyrimidine use, defined as: receiving an overdose of medication (total dose and/or infusion rate higher than recommended in the package insert) and/or Grade 3 or 4 serious adverse events after fluoropyrimidine exposure, according to CTCAE v5.0, which may include (but are not limited to): nausea, vomiting, diarrhea, anemia, neutropenia, febrile neutropenia, thrombocytopenia, and mucositis;
Lack of access to uridine triacetate (UT) in the standard of care;
Diagnosis of an invasive solid tumor under systemic treatment with a fluoropyrimidine (5-fluorouracil or capecitabine);
Organ function considered adequate by the investigator prior to the current fluoropyrimidine intoxication episode;
Ability to take oral medication;
For men and women of reproductive potential, agreement to practice abstinence or use highly effective contraceptive methods during study participation and for at least 6 months after the last dose of IP;
Men must agree not to donate sperm for at least 6 months after the last dose of IP;
Body surface area between 1.4 and 2.4 m², calculated using the Du Bois method;
AST/ALT within normal limits for participants without liver metastases;
AST/ALT up to 3x the upper limit of normal in participants with liver metastases;
Total and fractionated bilirubin up to 2x the upper limit of normal;
Agreement to abstain from alcohol consumption during the treatment period…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.