Trial Outcomes & Findings for NEO: Neoadjuvant Chemotherapy, Excision and Observation for Early Rectal Cancer (NCT NCT03259035)
NCT ID: NCT03259035
Last Updated: 2024-10-10
Results Overview
Defined as the percentage of patients with tumour downstaging to ypT0/T1good N0 and who avoid radical surgery.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
58 participants
Primary outcome timeframe
3 years
Results posted on
2024-10-10
Participant Flow
Participant milestones
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Stage 1: Chemotherapy
STARTED
|
58
|
|
Stage 1: Chemotherapy
COMPLETED
|
58
|
|
Stage 1: Chemotherapy
NOT COMPLETED
|
0
|
|
Stage 2: Tumor Excision
STARTED
|
56
|
|
Stage 2: Tumor Excision
COMPLETED
|
56
|
|
Stage 2: Tumor Excision
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
NEO: Neoadjuvant Chemotherapy, Excision and Observation for Early Rectal Cancer
Baseline characteristics by cohort
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Age, Continuous
|
66.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
41 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
48 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
54 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 yearsPopulation: All participants who had chemotherapy.
Defined as the percentage of patients with tumour downstaging to ypT0/T1good N0 and who avoid radical surgery.
Outcome measures
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Percentage of Organ Preservation
|
57 percentage of participants
Interval 45.0 to 68.0
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: All patients enrolled
Locoregional relapse is defined as reappearance of a tumour within the rectum or pelvis. The percentage of loco-reginal relapse at 3 years was estimated by Kaplan-Meier method for the survival function of the loco-regional relapse free survival, defined as the time from enrollment to the first date of definitive evidence (clinical, radiological or pathological) of locoregional relapses with patients who developed distant relapse only, died, loss to follow up, or were alive at clinical cut-off censored at respectively at last date of distant relapses, date of death, date of lost to follow-up, or last disease assessment date.
Outcome measures
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Percentage of Locoregional Relapses at 3 Years
|
7.1 percentage of participants
Interval 2.7 to 17.7
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: All participants enrolled
Distant relapse is defined as appearance of rectal cancer disease at sites remote from the rectum. The percentage of distant relapse at 3 years was estimated also by Kaplan-Meier method for the survival function of the distant free survival, defined as the time from enrollment to the first date of definitive evidence (clinical, radiological or pathological) of distant relapses with patients who died, loss to follow up, or were alive at clinical cut-off censored at respectively: date of death, date of lost to follow-up, and last disease assessment date.
Outcome measures
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Percentage of Distant Relapse at 3 Years
|
5.3 percentage of participants
Interval 1.7 to 15.4
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: All participants enrolled.
Percentage of disease free at 3 years was estimated by Kaplan-Meier method for the survival function of Disease-Free Survival (DFS), which is defined as the interval from date of enrollment to the first date of the events defined below: * Locoregional relapse * Distant relapse * Non-protocol radiotherapy, chemotherapy, or biologic therapy without documentation of the site of failure * Death due to any other reason. Patients who were alive without locoregional or distant relapse or receiving non-protocol radiotherapy, chemotherapy, or biologic therapy without documentation of the site of failure at the clinical data cutoff date were censored at their last disease assessment date. 3 year disease free survival was estimated by Kaplan-Meier method.
Outcome measures
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Percentage of Disease Free at 3 Years
|
84.3 percentage of participants
Interval 72.0 to 91.5
|
SECONDARY outcome
Timeframe: 1 dayPopulation: Patients who had BOTH chemotherapy and tumor excision with transanal endoscopic microsurgery (TEM) or transanal minimally invasive surgery (TAMIS)
Percentage of patients with at least one intraoperative injury
Outcome measures
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=53 Participants
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Rate of Intraoperative Complications
|
1.89 percentage of participants
|
Adverse Events
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
Serious events: 1 serious events
Other events: 58 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 participants at risk
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
1.7%
1/58 • During chemotherapy treatment (maximum 24 weeks)
|
Other adverse events
| Measure |
Chemotherapy (FOLFOX or CAPOX) Followed by Tumour Excision
n=58 participants at risk
FOLFOX infusion on a 14 day cycle (IV oxaliplatin 85 mg/m2 and leucovorin 400 mg/m2 over 2 hours on day 1 and bolus fluorouracil 400 mg/m2 over 5 - 15 minutes and continuous infusion of fluorouracil 2400 mg/m2 over 46 to 48 hours on days 1-2) for a total of 6 cycles or CAPOX on a 21 day cycle (IV oxaliplatin 130 mg/m2 over 2 hours on day 1 and 1000 mg/m2 capecitabine orally twice daily for 14 days from day 1) for a total of 4 cycles.
|
|---|---|
|
Eye disorders
Watering eyes
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Abdominal pain
|
19.0%
11/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Bloating
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Constipation
|
41.4%
24/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Diarrhea
|
55.2%
32/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Dyspepsia
|
13.8%
8/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Dysphagia
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Flatulence
|
12.1%
7/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
15.5%
9/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Mucositis oral
|
37.9%
22/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Nausea
|
72.4%
42/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Other gastrointestinal disorders
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
32.8%
19/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Rectal pain
|
19.0%
11/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Gastrointestinal disorders
Vomiting
|
22.4%
13/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
General disorders
Facial pain
|
10.3%
6/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
General disorders
Fatigue
|
77.6%
45/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
General disorders
Fever
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
General disorders
Non-cardiac chest pain
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
General disorders
Pain
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Infections and infestations
Skin infection
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Investigations
Weight loss
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Metabolism and nutrition disorders
Anorexia
|
41.4%
24/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Metabolism and nutrition disorders
Dehydration
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.8%
8/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Other musculoskeletal and connective tissue disorder
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
17.2%
10/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Dizziness
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Dysesthesia
|
27.6%
16/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Dysgeusia
|
20.7%
12/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Headache
|
17.2%
10/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Paresthesia
|
34.5%
20/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
63.8%
37/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Psychiatric disorders
Depression
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Psychiatric disorders
Insomnia
|
10.3%
6/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Renal and urinary disorders
Urinary frequency
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Renal and urinary disorders
Urinary retention
|
8.6%
5/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.7%
12/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.3%
6/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Laryngopharyngeal dysesthesia
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
10.3%
6/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
13.8%
8/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Other skin and subcutaneous tissue disorders
|
5.2%
3/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
27.6%
16/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
15.5%
9/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Vascular disorders
Flushing
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Vascular disorders
Hypertension
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
|
Vascular disorders
Thromboembolic event
|
6.9%
4/58 • During chemotherapy treatment (maximum 24 weeks)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place