CancerPhase 1/2ARCTURUSNCT07032142
Dose Optimization and Efficacy Assessment of a Fluoropyrimidine Antidote
A Phase 1/2 study testing I-01/23 in people with Cancer and Toxicity Due to Chemotherapy, run by D'Or Institute for Research and Education at 5 sites in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cancer, Toxicity Due to Chemotherapy
- What is being tested?
- I-01/23
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 5 sitesBrazil
- Study dates
- Jul 2025to Jul 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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;
- As a specific inclusion criterion for participants in Phase 1 of the study: a medical indication, according to routine care, for hospitalization of at least 48 hours for the clinical management of fluoropyrimidine-related toxicity.
Who cannot join
- Pregnant or breastfeeding women;
- Known history of allergic reaction to the molecules of the copound and/or to other molecules in the same class;
- Life expectancy of less than 30 days prior to hospital admission, based on underlying cancer and existing comorbidities;
- Estimated creatinine clearance <70 mL/min;
- Liver cirrhosis;
- Known liver or kidney disease;
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- Individuals with acquired immunodeficiency may be included only if they have no active opportunistic infections and following careful clinical assessment by the investigator, taking into account concurrent medications;
- Family history or known deficiency of the enzyme responsible for metabolizing the molecules of the copound and/or to other molecules in the same class;
- Uncontrolled infection;
- Hemodynamically unstable patients;
- Patients under orotracheal intubation;
- Patients unable to take oral medication;
- Prolonged QT interval;
- CNS metastases considered uncontrolled by the investigator;
- History of malabsorptive or inflammatory gastrointestinal disease;
- Use within the last 30 days of lactulose, protease inhibitors, amiodarone, carbamazepine, phenytoin, phenobarbital, oxcarbazepine, rifabutin, rifampin, or rifapentine;
- Use within the last 5 days of dietary supplements containing the molecules of the copound;
- Use within the last 30 days of drugs classified as anticonvulsants;
- Personal history of seizures;
- Comorbidities deemed limiting by the investigator;
- History of renal or liver transplant;
- Presence of intestinal obstruction.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Intituto D'Or de Pesquisa e Ensino São Paulo contact
5 sites in 1 country
Brazil5 sites
Instituto D'Or de Pesquisa e Ensino de Brasília
Brasília, Federal District
pesquisaclinica@idor.org, oncologia.projetos@idor.org
Instituto D'Or de Pesquisa e Ensino de Curitiba
Curitiba, Paraná
pesquisaclinica@idor.org, oncologia.projetos@idor.org
Instituto D'Or de Pesquisa e Ensino do Rio de Janeiro
Rio de Janeiro, Rio de Janeiro
pesquisaclinica@idor.org, oncologia.projetos@idor.org
Instituto D'Or de Pesquisa e Ensino de Salvador
Salvador, Estado de Bahia
pesquisaclinica@idor.org, oncologia.projetos@idor.org
Instituto D'Or de Pesquisa e Ensino de São Paulo
São Paulo, São Paulo
pesquisaclinica@idor.org, oncologia.projetos@idor.org
About this study
Official title: A Phase I/II Trial to Evaluation of the Dose and Efficacy of an Antidote to Fluoropyrimidines
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.
- Conditions
- Cancer, Toxicity Due to Chemotherapy
- Being tested
- I-01/23
- Keywords
- cancer, fluoropyrimidines, Toxicities from Fluoropyrimidines
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
I-01/23Drug
The I-01/23 will be administered over a 5-day period, with dosing adjusted according to the patient's body surface area (BSA).
| Study group | Role | What they receive |
|---|---|---|
| Patients who developed severe (grade 3 or higher) toxicities from the use of Fluoropyrimidines | Experimental | Not described Receives: Drug: I-01/23 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) Determination
Definition of DMT (Phase I) : The maximum tolerated dose (DMT) is the highest dose that produces the desired effect without resulting in unacceptable side effects
Measured: 6 days
Survival rate
The rate will be calculated as the percentage of participants alive following the initiation of treatment
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2025
- Study startJul 2025 (estimated)
- Primary completionJul 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- D'Or Institute for Research and Education
- Sponsor type
- University, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico
- Responsible party
- Principal investigator, Camila Motta Venchiarutti Moniz, D'Or Institute for Research and Education
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07032142
- Sponsor's protocol ID
- ARCTURUS 81626124.3.1001.5249
- Other IDs
- 81626124.3.1001.5249
- Acronym
- ARCTURUS
- Status last verified
- Apr 2025
- First submitted
- 21 May 2025
- Last updated
- 22 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial to Evaluation of the Dose and Efficacy of an Antidote to Fluoropyrimidines
- ClinicalTrials.gov ID
- NCT07032142
- Acronym / sponsor ID
- ARCTURUS / ARCTURUS 81626124.3.1001.5249
- Conditions
- Cancer, Toxicity Due to Chemotherapy
- Keywords
- cancer, fluoropyrimidines, Toxicities from Fluoropyrimidines
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- D'Or Institute for Research and EducationUniversity, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico
- Study start
- Jul 2025 (estimated)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 22 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients who developed severe (grade 3 or higher) toxicities from the use of Fluoropyrimidines | Experimental | Drug: I-01/23 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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;
Key exclusion criteria (22 listed)
- Pregnant or breastfeeding women;
- Known history of allergic reaction to the molecules of the copound and/or to other molecules in the same class;
- Life expectancy of less than 30 days prior to hospital admission, based on underlying cancer and existing comorbidities;
- Estimated creatinine clearance <70 mL/min;
- Liver cirrhosis;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| I-01/23The I-01/23 will be administered over a 5-day period, with dosing adjusted according to the patient's body surface area (BSA). | Drug | Patients who developed severe (grade 3 or higher) toxicities from the use of Fluoropyrimidines |
Primary outcome
Maximum Tolerated Dose (MTD) Determination
Time frame: 6 days
Definition of DMT (Phase I) : The maximum tolerated dose (DMT) is the highest dose that produces the desired effect without resulting in unacceptable side effects
Survival rate
Time frame: 7 days
The rate will be calculated as the percentage of participants alive following the initiation of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Intituto D'Or de Pesquisa e Ensino São Paulo contact
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing I-01/23 in people with Cancer and Toxicity Due to Chemotherapy, run by D'Or Institute for Research and Education at 5 sites in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Intituto D'Or de Pesquisa e Ensino São Paulo (pesquisaclinica@idor.org, oncologia.projetos@idor.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) Determination (time frame: 6 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2025.