New chemo combo may offer hope for rare, aggressive gut cancer
NCT ID NCT04325425
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether a chemotherapy regimen called mFOLFIRINOX works better than the standard platinum-etoposide combination for people with a rare, fast-growing type of neuroendocrine cancer that has spread. About 218 adults with this cancer will be randomly assigned to one of the two treatments. The goal is to see which regimen delays cancer growth longer and to find genetic markers that predict which treatment is best for each person.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 218 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2020
- Expected to finish
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Sep 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Grade 3 neuroendocrine carcinoma or high grade MiNEN with a grade 3 poorly differentiated neuroendocrine carcinoma component ≥30% of gastro-entero-pancreatic or unknown primary * Poorly differentiated * Small cell or large cell or non-small cell or non- typeable * Metastatic disease * First-line, no prior therapy for metastatic disease, no prior use of carboplatin, oxaliplatin, cisplatin, etoposide, irinotecan and 5-fluorouracile * At least one measurable lesion as assessed by CT-scan or MRI according to RECIST 1.1 guidelines * Available tumor block * ANC ≥ 1.5x109/l, platelet ≥ 100x109/l and hemoglobin \> 8 g/dl * Total bilirubin ≤ 1.5N, AST ≤ 2.5N, ALT≤ 2.5N or AST/ALT ≤ 5N in case of liver metastases. * Age ≥ 18 years * ECOG Performance Status ≤ 1 * Signed and dated informed consent, and willing and able to comply with protocol requirements. * Women of childbearing potential, as well as men (who have sexual relations with women of childbearing potential) must agree to use an effective method of contraception throughout this study and during the 15 months following administration of the last dose of the study medicinal product * Patient who is a beneficiary of the Social security system Exclusion Criteria: * Grade 3 well differentiated neuroendocrine tumor according to WHO 2017 classification * Severe renal impairment (creatinine clearance less than 30 mL/min, MDRD) * Partial or complete Dihydropyrimidine Dehydrogenase (DPD) deficiency (uracilemia ≥ 16 ng/mL) * Gilbert's syndrome * Pre-existing permanent neuropathy (NCI CTC V4.0 grade ≥2) * Previously treated by chemotherapy or targeted therapy * Brain metastases unless they are asymptomatic or under stable corticosteroid doses for 2 weeks otherwise. Radiation therapy prior to inclusion is required if symptomatic. * Combination with sorivudine and others analogues as brivudine (irreversibly inhibits the enzyme dihydropyrimidine dehydrogenase) * Treatment with St John's Wort (Hypericum perforatum) * Pregnant women or breastfeeding mother * Known or historical active infection with HIV, or known active infection untreated with hepatitis B or hepatitis C * History of prior malignancy, except for cured non-melanoma skin cancer, cured in situ cervical carcinoma, or other treated malignancies with no evidence of disease for at least three years. * Active or suspected acute or chronic uncontrolled disease that would impart, in the judgment of the investigator, excess risk associated with study participation or study drug administration, or which, in the judgment of the investigator, would make the patient inappropriate for entry into this study * vaccinations (live vaccine) within 30 days prior to start of study drugs * Patient under guardianship and/or deprived of his/her freedom * QT/QTc interval \> 450 msec for male and \> 470 msec for female at EKC. * K+ \< LLN, Mg²+ \< LLN, Ca²+ \< LLN * History or know hypersensitivity to any of the study chemotherapy agents, or their excipients
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
6 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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CH de Troyes
RECRUITINGTroyes, 10003, France
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Centre Hospitalier de Saint Malo
RECRUITINGSt-Malo, 35403, France
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Chu Dijon Bourgogne
RECRUITINGDijon, 21000, France
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Chu de Caen
RECRUITINGCaen, 14033, France
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Chu de Limoges - Dupuytren
RECRUITINGLimoges, 87042, France
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Institut de Cancérologie de Bourgogne
RECRUITINGDijon, 21000, France
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