Colorectal cancer (CRC) is the 2nd to 3rd most common malignant disease in developed countries, with over 1 million new cases and 500,000 deaths worldwide each year. The primary treatment for early stage CRC is surgery to remove the tumour, which is possible in 80% of patients. Even after surgery up to half of patients will develop recurrence or spread of the disease (metastases) which is incurable. Survival after 5 years is approximately 14% for patients with metastatic disease. Clinical trials using immunotherapy drugs called 'immune checkpoint inhibitors' have shown excellent results in advanced colorectal cancer patients who have certain genetic characteristics called 'mismatch repair deficiency (MMR-d)' and 'high microsatellite instability (MSI-h)'. The benefits of immunotherapy as a treatment prior to surgery to remove the tumour (neoadjuvant treatment) has been observed in both melanoma and in glioblastoma with enhanced local and systemic anti-tumour responses. Pembrolizumab is an immunotherapy drug and works by helping the body's own immune system to fight the cancer cells. The NEOPRISM-CRC trial will investigate whether giving pembrolizumab before surgery is safe, whether it improves the chances of the tumour being removed completely and whether it delays or prevents the cancer from coming back. Pembrolizumab treatment lasts for a maximum of 9 weeks (maximum of 3 cycles of treatment, each cycle consisting of 3 weeks) and is given prior to surgery. Following surgery patients will be followed up for at least 3 years after their surgery and to a maximum of 5 years. Target recruitment is 78 patients and recruitment is expected to take place over a 42 month period. Blood, tissue, mouth swabs and stool samples will be collected from patients throughout the trial to better understand the biology of immunotherapy as a treatment for CRC prior to surgery.
The NEOPRISM-CRC study is a phase II clinical trial. The purpose of the trial is to evaluate the efficacy and safety of pembrolizumab (Study IMP) in patients with high risk Stage 2 or Stage 3 MMR-deficient colorectal cancer stratified with tumour mutation burden status. Pembrolizumab is a type of immunotherapy that has demonstrated excellent results in clinical trials for patients with advanced colorectal cancer who have certain genetic characteristics (mismatch repair deficiency (MMR-d) / high microsatellite instability (MSI-H)). There are previous studies providing evidence that checkpoint inhibitors when given before surgery have shown better local and systemic anti-tumour responses in melanoma and glioblastoma than surgery alone. The main question the researcher wants to investigate is whether pembrolizumab given prior to having surgery delays or prevents the cancer from coming back in advanced colorectal cancer patients with MSI-H or MMR-d. The trial will investigate whether the treatment is safe, whether it improves pathological complete response rate and whether it improves the chances of the tumour being removed completely. The trial will also investigate whether pembrolizumab causes any delay to surgery due to side effects. The trial will also measure post-surgery complications and impact of the treatment on patient's quality of life. Once patients have enrolled into the NEOPRISM-CRC trial, if patient has consented to a trial colonoscopy, tissue samples will be taken during the procedure and shipped to the FoundationOne Medicine testing Laboratory and analysed to determine the TMB (tumour mutation burden) status assessed by the FOUNDATIONONE®CDx (FM1 Solid CDx) test. If patient does not consent to Trial Colonoscopy, blood sample will be taken to test the TMB status assessed by the FOUNDATIONONE® Liquid CDx (FM1 Liquid CDx) test . In addition if a patient has consented to Trial Colonoscopy, a PCR (Polymerase Chain reaction) test or Microsatellite testing will be done on tissue sample to confirm the Microsatellite Instability (MSI) status. Patients will receive one of two pre-operative regimens depending upon their TMB status/MSI status All patients will have one cycle of pembrolizumab 200 mg IV (a cycle is 21 days). Prior to cycle 2 the FM1 result should be available and patients will continue their treatment as follows: * If results is TMB-high or medium (or MSI-h if FM1 Solid CDx/ Liquid CDx test is not evaluable): A further two cycles of pembrolizumab 200 mg IV every 21 days and then will proceed to surgery 4 - 6 weeks after last dose of pembrolizumab * If result is TMB-low (or if FM1 Solid CDx/ Liquid CDx test and MSI result are not evaluable): No further cycles of pembrolizumab; will proceed to surgery 4 - 6 weeks after last dose of pembrolizumab Following surgery, patients will receive standard of care adjuvant treatment which may include post-operative chemotherapy in accordance with investigators clinical decision. Patients will continue to be followed up according to routine standard of care for a maximum of 5 years after surgery. Target recruitment is 78 patients and recruitment is expected to take place over a 42 month period.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
78
Dose: 200mg by IV infusion on Day 1 of each treatment cycle
Beatson West of Scotland Cancer Centre
Glasgow, United Kingdom
NOT_YET_RECRUITINGSt James University Hospital (SJUH)
Leeds, United Kingdom
RECRUITINGUniversity College Hospital
London, United Kingdom
RECRUITINGChristie Hospital NHS Trust, Wilmslow Road,
Manchester, United Kingdom
RECRUITINGSouthampton General Hospital
Southampton, United Kingdom
RECRUITINGRelapse-free survival
Relapse-free Survival (RFS) is defined as time from start of treatment to time of any signs or symptoms of the cancer or time of death from any cause.
Time frame: From registration to any signs or symptoms of the cancer or death assessed up to a maximum of 3 years from surgery date
Pathological complete response rate (pCR)
A pCR will be defined as having no residual cancer cells in the resected specimen at surgery. Patients who do not achieve pCR or who do not proceed to resectional surgery for any reason will be counted as non-responders.
Time frame: Pathological complete response rate (pCR) assessed at 3 months following surgery
Overall survival
Overall survival (OS) is defined as the time from start of treatment to time of death from any cause.
Time frame: From registration to death from any cause, assessed up to a maximum of 3 years from surgery date
Frequency and severity of adverse events
Adverse events recorded continuously in relation to each treatment cycle graded using CTCAE criteria
Time frame: From informed consent to 3 months after surgery
Rate of R0 resection and completed surgery
The incidence of resection types (R0, R1 and R2) will be presented in tables using frequencies, as well as the rate of surgery completed.
Time frame: Assessed from surgery to 28-35 days following surgery
Frequency and severity of post-operative surgical complications
The incidence of surgical complications will be described in tables by complication type/grade using frequencies using the Clavien-Dindo grading system.
Time frame: Assessed from surgery up to last follow up visit (maximum of 5 years from date of surgery)
Health-related Quality of Life (QoL) and functional outcome
The European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) will be used. The EORTC QLQ-C30 comprises 30 items (i.e. single questions), 24 of which are aggregated into nine multi-item scales, that is, five functioning scales (physical, role, cognitive, emotional and social), three symptom scales (fatigue, pain and nausea/vomiting) and one global health status scale. The remaining six single-item (dyspnoea, appetite loss, sleep disturbance, constipation, diarrhoea and the financial impact) scales assess symptoms. All of the scales and single-item measures range in score from 0 to 100. Higher score for the functioning scales and global health status denote a better level of functioning (i.e. a better state of the patient), while higher scores on the symptom and single-item scales indicate a higher level of symptoms (i.e. a worse state of the patient).
Time frame: From informed consent to 28-35 days following surgery
Health-related Quality of Life (QoL) and functional outcome
The EuroQol- 5 Dimension (EQ-5D) will be used. Each dimension in the EQ-5D has five response levels: no problems (Level 1); slight; moderate; severe; and extreme problems (Level 5). Lower score indicates better health, whereas higher score indicate more severe or frequent problems.
Time frame: From informed consent to 28-35 days following surgery
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