Rectum CancerBJCC-R01NCT04405206
Total Neoadjuvant Therapy Followed by 'Watch and Wait' Approach or Organ Preservation for Low-risk Rectal Cancer
An observational study of Rectum Cancer, run by Peking University Cancer Hospital & Institute at 1 site in China.
Trial summary
Aim: To investigate the safety and efficacy of organ preservation (OP) with watch-and-wait strategy (W\&W) or local excision (LE) in MRI stratified low-risk rectal cancer treated by total neoadjuvant treatment. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Rectum Cancer
- What is being tested?
- Oxaliplatin, Capecitabine and intensity modulated radiotherapy
- Type of treatment
- Surgery or procedure, Lifestyle or therapy programme
- How many people
- 54 participants
- Where
- 1 siteChina
- Study dates
- 25 May 2020to 30 Dec 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Rectal cancer patient who receives primary care in Beijing Cancer Hospital will be selected as the candidate for this study.
Who may be able to join
- 1. the age is more than 18 years old and less than 85 years old;
- 2. ECOG score of physical condition is 0-1;
- 3. adenocarcinoma of rectum confirmed by pathology; differentiated into Grade 1-3, i.e. high, medium and low differentiated adenocarcinoma
- 4. the distance from the lower edge of the tumor to the anal edge ≤ 12cm (endoscopy) or to the anorectal ring (ARJ) ≤ 8cm;
- 5. the initial MRI was T2 / T3a / T3B, with negative EMVI and negative CRM. There was no lymph node metastasis outside the ilium, general ilium, obturator and abdominal aorta
- 6. the maximum diameter of tumor ≤ 4cm or the circumferentially invasive area is less than 1 / 3 of intestinal circumference
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- 7. no evidence of distant metastasis;
- 8. no history of pelvic radiotherapy;
- 9. no history of surgery or chemotherapy for rectal cancer;
- 10. systemic infection without antibiotic treatment;
- 11. blood routine test: neutrophil absolute value > 1.5 × 10 9 / L, HGB > 10.0 g / dl, PLT > 100 × 10 9 / L;
- 12. blood biochemistry: total bilirubin ≤ 1.5 x ULN, AST ≤ 3 x ULN, ALT ≤ 4 x ULN;
- 13. the patient read and signed the informed consent of the study and agreed to participate in the study;
Who cannot join
- 1. recurrent rectal cancer;
- 2. initial local non resectable rectal cancer (no possibility of R0 whole block resection);
- 3. the creatinine level is 1.5 times higher than the upper limit of normal value;
- 4. have a history of pelvic radiotherapy;
- 5. the patients could not tolerate the enhanced MRI;
- 6. malignant tumors whose survival rate in the past 5 years is significantly lower than that of rectal cancer in our center (excluding well treated basal cell carcinoma, skin squamous carcinoma, small renal carcinoma, breast cancer and thyroid papillary carcinoma);
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- 7. in the past 6 months, the patients had arterial embolism diseases, such as angina, MI, TIA, CVA, etc;
- 8. have received other types of anti-tumor or experimental treatment;
- 9. the patients are pregnant or lactating women;
- 10. patients with other diseases or mental disorders may affect their participation in this study;
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Beijing Cancer Hospital
Beijing, 100142
Yiming Zhao, M.D., +8618600490721, liyingjiedr@sina.com
Aiwen Wu, M.D.
About this study
Official title: Total Neoadjuvant Therapy Followed by 'Watch and Wait' Approach or Organ Preservation for MRI Stratified Low-risk Rectal Cancer: a Multi-center, Prospective, Single-arm Phase II Trial.
Aim: To investigate the safety and efficacy of organ preservation (OP) with watch-and-wait strategy (W\&W) or local excision (LE) in MRI stratified low-risk rectal cancer treated by total neoadjuvant treatment. Meanwhile we will look into the role of ctDNA in the prediction of regrowth and metastasis in the wait and wait process.
Methods: Low-risk rectal cancer with following MRI features are recruited: mid-low tumor, mrT2-3b, MRF(-), EMVI(-), differentiation grade 1-3. Patients will receive IMRT 50.6Gy/22f with concurrent capecitabine and 4 cycles of consolidation CAPEOX. Patients with cCR/near-cCR were recommended for 'watch \& wait' approach or local excision (LE). The OPR and sphincter preservation rate (SPR) at 2 years will be analyzed.
As the extension of PKUCH-R01, BJCC-R01 trial will upgrade to a multi-center research enrolled 3 other colorectal center in Beijing.
Read the detailed description
Neoadjuvant chemoradiotherapy (nCRT), total mesorectal excision and adjuvant chemotherapy comprise the standard treatment for locally advanced rectal cancer, following which 15-30% patients achieved pathological complete response need to receive the removal of rectum without residual tumor and suffer significant functional impairment even after sphincter preservation. Adjuvant chemotherapy is also questioned for its benefit for prolonged survival through the data from various studies. More evidence demonstrated that organ-preservation (e.g. non-operative management or local excision) for patients with clinical complete response (cCR) or near-cCR following nCRT had similar survival when compared with those received standard care.
This study is designed to investigate the efficacy of neoadjuvant intensity modulated nCRT with concurrent capecitabine plus consolidation CapeOX for T2/DWI/Enhanced MRI defined cT2-T3b mid-low rectal cancer without threatening mesorectal fascia or extramural vascular invasion (EMVI) or mrN2 disease.
According to the response to treatment evaluated by multi-modal assessment including digital exam, T2/DWI/Enhanced MRI, endoscopy and serum CEA test, patients will receive tailored operative management like local excision or total mesorectal excision, or non-operative management. Intention to treatment was also allowed in this study.
Firstly, the investigators will observe the organ preservation rate at 2 years. Endpoints for organ-preservation like non-regrowth DFS, stoma-free survival and other conventional survival outcomes (DFS, OS) would be further collected. The short-term and long-term QoL will be measured in all patients.
. Our baseline data showed the 48% of locally advanced rectal cancers could be downstaged to stage ypT0-2N0 following IMRT with concurrent capecitabine. We hypothesize that at least 24% of rectal cancers could be candidates for LE or NOM after IMRT and the rectum preservation rate will increase to 40% in low-risk rectal cancers by LE or NOM following IMRT plus consolidation CapeOX at 2 years. As a superiority design, this study need to recruit 64 patients to test this hypothesis, with 85% power (exact binomial test for proportions, alpha = 5 %, one-sided), If the number of responses is 22 or more, the hypothesis that P <= 0.240 is rejected. We anticipate about 10 % loss to follow-up, so we will recruit an additional 8 patients and the study will recruit 72 patients in all.
- Conditions
- Rectum Cancer
- Being tested
- Oxaliplatin, Capecitabine, intensity modulated radiotherapy, DRE-Endoscopy-MRI-CEA, Nonoperative Management
- Keywords
- rectum cancer
- Medical subject headings
- Rectal Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OxaliplatinDrug
Also called OXA
OXA 130mg/m2
CapecitabineDrug
Also called Cape
Cape 1000mg/m2
intensity modulated radiotherapyRadiation
Also called IMRT
IMRT 50.4Gy/22f
DRE-Endoscopy-MRI-CEAProcedure or surgery
examination process in follow up
Nonoperative ManagementProcedure or surgery
Also called NOM
Watch and Wait
Local excisionProcedure or surgery
Also called LE
For ymriT1N0 and near-cCR patients，LE is optional.
Total Mesorectal ExcisionProcedure or surgery
Also called TME
Standard TME surgery openly or laporoscopically
Quality of Life QuestionnairesBehavioural
Also called QLQ
QLQ C30 and QLQ CR29
| Study group | Role | What they receive |
|---|---|---|
| Group A:Clinical complete response (cCR) | Group A:Clinical complete response (cCR) Patients who achieve cCR when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation CapeOX(Capecitabine+Oxaliplatin) will receive Nonoperative Management(NOM). Receives: Drug: Oxaliplatin; Drug: Capecitabine; Radiation: intensity modulated radiotherapy; Procedure: DRE-Endoscopy-MRI-CEA; Procedure: Nonoperative Management; Behavioral: Quality of Life Questionnaires | |
| Group B:Near-cCR | Patients who achieve near-cCR when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation CapeOX(Capecitabine+Oxaliplatin) will receive Local Excision(LE) or Nonoperative Management(NOM). Receives: Drug: Oxaliplatin; Drug: Capecitabine; Radiation: intensity modulated radiotherapy; Procedure: DRE-Endoscopy-MRI-CEA; Procedure: Nonoperative Management; Procedure: Local excision; Behavioral: Quality of Life Questionnaires | |
| Group C:Residual tumor | Patients with residual tumor when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation Receives: Drug: Oxaliplatin; Drug: Capecitabine; Radiation: intensity modulated radiotherapy; Procedure: DRE-Endoscopy-MRI-CEA; Procedure: Total Mesorectal Excision; Behavioral: Quality of Life Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
OPR(organ preservation rate)
Proportion of patients who achieved cCR or near cCR after neoadjuvant treatment and received watch and wait approach or local excision
Measured: 3 year
Secondary outcomes (2)
NR-DFS(Non-regrowth disease free survival)
Within 3 years after receiving the neoadjuvant treatment, the time patient in status free of death, local recurrence after radical resection or distant metastasis.
Measured: 3 year
SFS(stoma-free survival)
Within 3 years after receiving the neoadjuvant treatment, the time patient in status free of stoma
Measured: 3 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2020
- Study start25 May 2020
- Primary completion30 Dec 2023 (estimated)
- Study completion30 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rectum CancerStudies of the same condition, open and under way.
- More trials run by Peking University Cancer Hospital & InstituteThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University Cancer Hospital & Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Aiwen Wu, Peking University Cancer Hospital & Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04405206
- Sponsor's protocol ID
- BJCC-R01
- Acronym
- BJCC-R01
- Status last verified
- Oct 2020
- First submitted
- 23 May 2020
- Last updated
- 14 Oct 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Total Neoadjuvant Therapy Followed by 'Watch and Wait' Approach or Organ Preservation for MRI Stratified Low-risk Rectal Cancer: a Multi-center, Prospective, Single-arm Phase II Trial.
- ClinicalTrials.gov ID
- NCT04405206
- Acronym / sponsor ID
- BJCC-R01 / BJCC-R01
- Conditions
- Rectum Cancer
- Keywords
- rectum cancer
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- Peking University Cancer Hospital & InstituteUniversity, hospital or other
- Study start
- 25 May 2020 (actual)
- Primary completion
- 30 Dec 2023 (estimated)
- Study completion
- 30 Dec 2023 (estimated)
- First posted
- 28 May 2020 (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.
Aim: To investigate the safety and efficacy of organ preservation (OP) with watch-and-wait strategy (W\&W) or local excision (LE) in MRI stratified low-risk rectal cancer treated by total neoadjuvant treatment. Meanwhile we will look into the role of ctDNA in the prediction of regrowth and metastasis in the wait and wait process.
Methods: Low-risk rectal cancer with following MRI features are recruited: mid-low tumor, mrT2-3b, MRF(-), EMVI(-), differentiation grade 1-3. Patients will receive IMRT 50.6Gy/22f with concurrent capecitabine and 4 cycles of consolidation CAPEOX. Patients with cCR/near-cCR were recommended for 'watch \& wait' approach or local excision (LE). The OPR and sphincter preservation rate (SPR) at 2 years will be analyzed.
As the extension of PKUCH-R01, BJCC-R01 trial will upgrade to a multi-center research enrolled 3 other colorectal center in Beijing.
Detailed description
Neoadjuvant chemoradiotherapy (nCRT), total mesorectal excision and adjuvant chemotherapy comprise the standard treatment for locally advanced rectal cancer, following which 15-30% patients achieved pathological complete response need to receive the removal of rectum without residual tumor and suffer significant functional impairment even after sphincter preservation. Adjuvant chemotherapy is also questioned for its benefit for prolonged survival through the data from various studies. More evidence demonstrated that organ-preservation (e.g. non-operative management or local excision) for patients with clinical complete response (cCR) or near-cCR following nCRT had similar survival when compared with those received standard care.
This study is designed to investigate the efficacy of neoadjuvant intensity modulated nCRT with concurrent capecitabine plus consolidation CapeOX for T2/DWI/Enhanced MRI defined cT2-T3b mid-low rectal cancer without threatening mesorectal fascia or extramural vascular invasion (EMVI) or mrN2 disease.
According to the response to treatment evaluated by multi-modal assessment including digital exam, T2/DWI/Enhanced MRI, endoscopy and serum CEA test, patients will receive tailored operative management like local excision or total mesorectal excision, or non-operative management. Intention to treatment was also allowed in this study.
Firstly, the investigators will observe the organ preservation rate at 2 years. Endpoints for organ-preservation like non-regrowth DFS, stoma-free survival and other conventional survival outcomes (DFS, OS) would be further collected. The short-term and long-term QoL will be measured in all patients.
. Our baseline data showed the 48% of locally advanced rectal cancers could be downstaged to stage ypT0-2N0 following IMRT with concurrent capecitabine. We hypothesize that at least 24% of rectal cancers could be candidates for LE or NOM after IMRT and the rectum preservation rate will increase to 40% in low-risk rectal cancers by LE or NOM following IMRT plus consolidation CapeOX at 2 years. As a superiority design, this study need to recruit 64 patients to test this hypothesis, with 85% power (exact binomial test for proportions, alpha = 5 %, one-sided), If the number of responses is 22 or more, the hypothesis that P <= 0.240 is rejected. We anticipate about 10 % loss to follow-up, so we will recruit an additional 8 patients and the study will recruit 72 patients in all.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group A:Clinical complete response (cCR) | – | Drug: Oxaliplatin, Drug: Capecitabine, Radiation: intensity modulated radiotherapy, Procedure: DRE-Endoscopy-MRI-CEA, Procedure: Nonoperative Management, Behavioral: Quality of Life QuestionnairesGroup A:Clinical complete response (cCR) Patients who achieve cCR when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation CapeOX(Capecitabine+Oxaliplatin) will receive Nonoperative Management(NOM). |
| Group B:Near-cCR | – | Drug: Oxaliplatin, Drug: Capecitabine, Radiation: intensity modulated radiotherapy, Procedure: DRE-Endoscopy-MRI-CEA, Procedure: Nonoperative Management, Procedure: Local excision, Behavioral: Quality of Life QuestionnairesPatients who achieve near-cCR when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation CapeOX(Capecitabine+Oxaliplatin) will receive Local Excision(LE) or Nonoperative Management(NOM). |
| Group C:Residual tumor | – | Drug: Oxaliplatin, Drug: Capecitabine, Radiation: intensity modulated radiotherapy, Procedure: DRE-Endoscopy-MRI-CEA, Procedure: Total Mesorectal Excision, Behavioral: Quality of Life QuestionnairesPatients with residual tumor when restaged by DRE-Endoscopy-MRI-CEA at 16 weeks following intensity modulated radiotherapy(IMRT) plus consolidation |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Rectal cancer patient who receives primary care in Beijing Cancer Hospital will be selected as the candidate for this study.
Key inclusion criteria (13 listed)
- 1. the age is more than 18 years old and less than 85 years old;
- 2. ECOG score of physical condition is 0-1;
- 3. adenocarcinoma of rectum confirmed by pathology; differentiated into Grade 1-3, i.e. high, medium and low differentiated adenocarcinoma
- 4. the distance from the lower edge of the tumor to the anal edge ≤ 12cm (endoscopy) or to the anorectal ring (ARJ) ≤ 8cm;
- 5. the initial MRI was T2 / T3a / T3B, with negative EMVI and negative CRM. There was no lymph node metastasis outside the ilium, general ilium, obturator and abdominal aorta
Key exclusion criteria (10 listed)
- 1. recurrent rectal cancer;
- 2. initial local non resectable rectal cancer (no possibility of R0 whole block resection);
- 3. the creatinine level is 1.5 times higher than the upper limit of normal value;
- 4. have a history of pelvic radiotherapy;
- 5. the patients could not tolerate the enhanced MRI;
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 |
|---|---|---|
| OxaliplatinAlso: OXAOXA 130mg/m2 | Drug | Group A:Clinical complete response (cCR), Group B:Near-cCR, Group C:Residual tumor |
| CapecitabineAlso: CapeCape 1000mg/m2 | Drug | Group A:Clinical complete response (cCR), Group B:Near-cCR, Group C:Residual tumor |
| intensity modulated radiotherapyAlso: IMRTIMRT 50.4Gy/22f | Radiation | Group A:Clinical complete response (cCR), Group B:Near-cCR, Group C:Residual tumor |
| DRE-Endoscopy-MRI-CEAexamination process in follow up | Procedure or surgery | Group A:Clinical complete response (cCR), Group B:Near-cCR, Group C:Residual tumor |
| Nonoperative ManagementAlso: NOMWatch and Wait | Procedure or surgery | Group A:Clinical complete response (cCR), Group B:Near-cCR |
| Local excisionAlso: LEFor ymriT1N0 and near-cCR patients，LE is optional. | Procedure or surgery | Group B:Near-cCR |
| Total Mesorectal ExcisionAlso: TMEStandard TME surgery openly or laporoscopically | Procedure or surgery | Group C:Residual tumor |
| Quality of Life QuestionnairesAlso: QLQQLQ C30 and QLQ CR29 | Behavioural | Group A:Clinical complete response (cCR), Group B:Near-cCR, Group C:Residual tumor |
Primary outcome
OPR(organ preservation rate)
Time frame: 3 year
Proportion of patients who achieved cCR or near cCR after neoadjuvant treatment and received watch and wait approach or local excision
2 secondary outcomes
NR-DFS(Non-regrowth disease free survival)
Time frame: 3 year
SFS(stoma-free survival)
Time frame: 3 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2020.
- Individual participant dataSharing planned: Undecided
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Rectum Cancer, run by Peking University Cancer Hospital & Institute at 1 site in China.
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 10 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: status not confirmed, as of the sponsor's last update on 14 Oct 2020. 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 China. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yiming Zhao, M.D. (+8618600490721 ext. 18600490721, iammike0721@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
OPR(organ preservation rate) (time frame: 3 year). 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 14 Oct 2020.