Rectal CancerPhase 2NCT04380337
Organ Preservation Program Using Short-Course Radiation & FOLFOXIRI in Rectal Cancer
A Phase 2 study testing FOLFOXIRI, FOLFOX regimen and XELOX in people with Rectal Cancer, run by Stanford University at 1 site in United States.
Trial summary
The purpose of the research is to evaluate whether both chemotherapy and radiotherapy can lead to higher rates of clinical complete response leading to organ preservation in human subjects with cancer. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Rectal Cancer
- What is being tested?
- FOLFOXIRI, FOLFOX regimen and XELOX
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 21 May 2020to 17 Jan 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- 1. Pathologically (histologically or cytologically) proven diagnosis of adenocarcinoma of rectum requiring total mesorectal excision as deemed by multidisciplinary evaluation
- 2.At least 18 years of age
- 3.For women of childbearing potential or who are not postmenopausal (see Appendix B for Definition of Menopausal Status), a negative urine or serum pregnancy test must be done. Also, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation and for up to 4 weeks following the study. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- 4.ECOG 0, 1, or 2
- 5.Ability to understand and the willingness to personally sign the written IRB approved informed consent document.
- 6.Patients must have acceptable organ and marrow function as defined below:
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- Absolute neutrophil count (ANC) >1,500/uL
- Hg > 8.0 g/dL; if blood transfusion is performed for achieving adequate hemoglobin level, the level should stay above goal for at least 1 week after transfusion
- Platelets >100,000/uL
- Total bilirubin <1.5X normal institutional limits
- aspartate aminotransferase (AST) (SGOT) / alanine aminotransferase (ALT)(SGPT) < 3X upper limit of normal
- Creatinine <1.5X upper limit of normal or creatinine clearance (CrCL)>50 by Cockcroft-Gault
- 7 Clinical stage >T2N0 or low T2N0 rectal cancer (AJCC, 8th ed.) including no metastases based on the following diagnostic workup:
- General history and physical examination with DRE (if deemed appropriate by treating physician) within 45 days prior to enrollment
- Sigmoidoscopy within 90 days prior to enrollment
- The following imaging studies are required within 45 days prior to enrollment:
- CT chest/abdomen/pelvis
- MRI Pelvis
Who cannot join
- 1.Prior pelvic RT or chemotherapy for rectal cancer.
- 2.Upper T2N0 rectal cancers eligible for sphincter-preservation surgery
- 3.Use of other investigational agents.
- 4.Ongoing or active infections requiring systemic antibiotic treatment or uncontrolled intercurrent illness including but not limited to symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- 5.Any concurrent malignancy other than non-melanoma skin cancer or carcinoma in situ of the cervix. Patients with any previous malignancy without evidence of disease for >3 years will be allowed to enter the trial.
- 6.Known hypersensitivity to 5-FU compounds.
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- 7.Pregnant and breastfeeding women are excluded. Women of child-bearing potential who are unwilling or unable to use an acceptable method of birth control to avoid pregnancy for the entire study period and for up to 4 weeks after the study are excluded. (This applies to women who have experienced menarche and have not undergone successful surgical sterilization or are not postmenopausal).
- Because patients with immune deficiency are at increased risk of lethal infections when treated with marrow-suppressive therapy, known HIV-positive patients with detectable viral loads and/or receiving combination anti-retroviral therapy are excluded from the study.
- 8.Primary unresectable rectal cancer (tumor invading adjacent organs and en bloc resection will not achieve negative margins).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Trial of Organ Preservation Program Using Short-Course Radiation and FOLFOXIRI for Rectal Cancer (SHORT-FOX)
The purpose of the research is to evaluate whether both chemotherapy and radiotherapy can lead to higher rates of clinical complete response leading to organ preservation in human subjects with cancer. The objective is to learn if this treatment approach may safely be used as an alternative to the standard treatment for rectal cancer and to know the quality-of-life in these patients.
Read the detailed description
Primary Objective: To assess clinical complete response of an organ preservation approach using short course radiation followed by intensified chemotherapy.
Secondary Objective: To assess safety in all enrolled patients, local regrowth rate and other cancer specific outcomes (metastasis-free survival, colostomy-free survival and overall survival), longitudinal health-related quality of life of this organ preservation approach
- Conditions
- Rectal Cancer
- Being tested
- FOLFOXIRI, FOLFOX regimen, XELOX, IMRT
- Medical subject headings
- Rectal Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FOLFOXIRIDrug
Chemotherapy regimen of Oxaliplatin 85mg/m2 , Leucovorin 400 mg/m2 , Irinotecan 165mg/m2 , 5-Fluorouracil
FOLFOX regimenDrug
Chemotherapy regimen of Oxaliplatin 85mg/m2, Leucovorin 400 mg/m2, 5-Fluorouracil 2400mg/m2
XELOXDrug
Chemotherapy regimen of Oxaliplatin 130 mg/m2, Capecitabine 1000mg/m2
IMRTRadiation
Also called Intensity-modulated radiotherapy (IMRT)
Radiotherapy (5 Gy x 5 fractions) with an additional boost fraction (5 Gy x 1 fraction) delivered sequentially
| Study group | Role | What they receive |
|---|---|---|
| Radiation/FOLFOXIRI | Experimental | Treatment will comprise 6 daily fractions of radiotherapy at 5 Gy per fraction followed by 4 months of FOLFOXIRI. Patients who have performance status or conditions that may preclude use of FOLFOXIRI may be treated with FOLFOX or XELOX. Those who achieve a clinical complete response will be considered for organ preservation approach. All other patients will receive standard of care total mesorectal excision (TME). Receives: Drug: FOLFOXIRI; Drug: FOLFOX regimen; Drug: XELOX; Radiation: IMRT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Complete Response (cCR)
Proportion of patients who achieve a clinical complete response following therapy, expressed as a number and proportion without dispersion.
Measured: 8 (+/-4 ) weeks following completion of RT and chemotherapy, an average of 9 months
Secondary outcomes (5)
Number of Participants With Toxicity
Toxicity defined as non-hematologic grade 4 adverse events using CTCAE v5 or higher toxicity at least possibly related to treatment, and assessed up to 3 months after completion of radiotherapy (RT) and chemotherapy.
Measured: 8 (+/- 4) weeks following completion of RT and chemotherapy, an average of 3 months
Local Regrowth Rate
Local regrowth is defined as the presence of adenocarcinoma within the rectal wall or within the mesorectum confirmed by pathology, expressed as a percentage with its 95% confidence interval.
Measured: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Disease Free Survival (DFS)
DFS defined as the time from the start date of RT to the date of the first documented progression or death due to any cause assessed up to 2 years after completion of chemotherapy. Patients will be censored at the last date of follow-up if lost to follow-up prior to two years, expressed as a median with interquartile range.
Measured: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Colostomy-free Survival
Colostomy-free survival defined as the time from the start date of RT to the date of colostomy or death due to any cause assessed up to 2 years after completion of chemotherapy, expressed as a median with interquartile range. Patients will be censored at the last date of follow-up if lost to follow-up prior to two years.
Measured: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Overall Survival (OS)
OS defined as death from any cause from start date of RT until death, study completion, or loss to follow-up, whichever occurs first. This will be reported as median survival time with interquartile range.
Measured: 27 (+/- 4) months following completion of RT and chemotherapy, an average of 2.25 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2020
- Study start21 May 2020
- Primary completion17 Jan 2024
- Study completion17 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Rectal CancerStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Erqi Liu Pollom, Stanford University
- Investigators
- Erqi L Pollom, Principal investigator, Stanford Universiy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04380337
- Sponsor's protocol ID
- IRB-56027
- Other IDs
- IRB-56027, COR0019
- Status last verified
- Feb 2025
- First submitted
- 5 May 2020
- Last updated
- 25 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Brown E, Fisher GA Jr, Shelton A, Chang DT, Pollom E. Advancing clinical trial equity through integration of telehealth and decentralized treatment. JNCI Cancer Spectr. 2024 Jul 1;8(4):pkae050. doi: 10.1093/jncics/pkae050.
PubMed 38902952
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Organ Preservation Program Using Short-Course Radiation and FOLFOXIRI for Rectal Cancer (SHORT-FOX)
- ClinicalTrials.gov ID
- NCT04380337
- Acronym / sponsor ID
- IRB-56027
- Conditions
- Rectal Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- 21 May 2020 (actual)
- Primary completion
- 17 Jan 2024 (actual)
- Study completion
- 17 Jan 2024 (actual)
- First posted
- 8 May 2020 (actual)
- Results
- Posted 25 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the research is to evaluate whether both chemotherapy and radiotherapy can lead to higher rates of clinical complete response leading to organ preservation in human subjects with cancer. The objective is to learn if this treatment approach may safely be used as an alternative to the standard treatment for rectal cancer and to know the quality-of-life in these patients.
Detailed description
Primary Objective: To assess clinical complete response of an organ preservation approach using short course radiation followed by intensified chemotherapy.
Secondary Objective: To assess safety in all enrolled patients, local regrowth rate and other cancer specific outcomes (metastasis-free survival, colostomy-free survival and overall survival), longitudinal health-related quality of life of this organ preservation approach
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Radiation/FOLFOXIRI | Experimental | Drug: FOLFOXIRI, Drug: FOLFOX regimen, Drug: XELOX, Radiation: IMRTTreatment will comprise 6 daily fractions of radiotherapy at 5 Gy per fraction followed by 4 months of FOLFOXIRI. Patients who have performance status or conditions that may preclude use of FOLFOXIRI may be treated with FOLFOX or XELOX. Those who achieve a clinical complete response will be considered for organ preservation approach. All other patients will receive standard of care total mesorectal excision (TME). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 1. Pathologically (histologically or cytologically) proven diagnosis of adenocarcinoma of rectum requiring total mesorectal excision as deemed by multidisciplinary evaluation
- 2.At least 18 years of age
- 3.For women of childbearing potential or who are not postmenopausal (see Appendix B for Definition of Menopausal Status), a negative urine or serum pregnancy test must be done. Also, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation and for up to 4 weeks following the study. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- 4.ECOG 0, 1, or 2
- 5.Ability to understand and the willingness to personally sign the written IRB approved informed consent document.
Key exclusion criteria (9 listed)
- 1.Prior pelvic RT or chemotherapy for rectal cancer.
- 2.Upper T2N0 rectal cancers eligible for sphincter-preservation surgery
- 3.Use of other investigational agents.
- 4.Ongoing or active infections requiring systemic antibiotic treatment or uncontrolled intercurrent illness including but not limited to symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- 5.Any concurrent malignancy other than non-melanoma skin cancer or carcinoma in situ of the cervix. Patients with any previous malignancy without evidence of disease for >3 years will be allowed to enter the trial.
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 |
|---|---|---|
| FOLFOXIRIChemotherapy regimen of Oxaliplatin 85mg/m2 , Leucovorin 400 mg/m2 , Irinotecan 165mg/m2 , 5-Fluorouracil | Drug | Radiation/FOLFOXIRI |
| FOLFOX regimenChemotherapy regimen of Oxaliplatin 85mg/m2, Leucovorin 400 mg/m2, 5-Fluorouracil 2400mg/m2 | Drug | Radiation/FOLFOXIRI |
| XELOXChemotherapy regimen of Oxaliplatin 130 mg/m2, Capecitabine 1000mg/m2 | Drug | Radiation/FOLFOXIRI |
| IMRTAlso: Intensity-modulated radiotherapy (IMRT)Radiotherapy (5 Gy x 5 fractions) with an additional boost fraction (5 Gy x 1 fraction) delivered sequentially | Radiation | Radiation/FOLFOXIRI |
Primary outcome
Clinical Complete Response (cCR)
Time frame: 8 (+/-4 ) weeks following completion of RT and chemotherapy, an average of 9 months
Proportion of patients who achieve a clinical complete response following therapy, expressed as a number and proportion without dispersion.
5 secondary outcomes
Number of Participants With Toxicity
Time frame: 8 (+/- 4) weeks following completion of RT and chemotherapy, an average of 3 months
Local Regrowth Rate
Time frame: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Disease Free Survival (DFS)
Time frame: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Colostomy-free Survival
Time frame: 22 (+/- 2) months following completion of RT and chemotherapy, an average of 24 months (2 years)
Overall Survival (OS)
Time frame: 27 (+/- 4) months following completion of RT and chemotherapy, an average of 2.25 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38902952Brown E, Fisher GA Jr, Shelton A, Chang DT, Pollom E. Advancing clinical trial equity through integration of telehealth and decentralized treatment. JNCI Cancer Spectr. 2024 Jul 1;8(4):pkae050. doi: 10.1093/jncics/pkae050.
- 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. This study is not recruiting at the moment.
Erqi L Pollom principal investigator
Stanford Universiy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FOLFOXIRI, FOLFOX regimen and XELOX in people with Rectal Cancer, run by Stanford University at 1 site in United States.
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 18 inclusion and 9 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: finished, as of the sponsor's last update on 25 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 phase mean?
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?
Yes. Results are posted in the registry (first posted 25 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Complete Response (cCR) (time frame: 8 (+/-4 ) weeks following completion of RT and chemotherapy, an average of 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04380337, which also lists 1 linked publication.
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 25 Feb 2025.