A Phase I clinical trial that will investigate the safety and tolerability of combining the modified vaccinia virus BT-001 with systemic pembrolizumab in patients with localised pMMR rectal cancer
Histological diagnosis of primary, localised rectal adenocarcinoma (cT2N0M0 to cT3bN2M0, TNM classification version 8
Diagnosis of Proficient Mismatch Repair (pMMR) rectal adenocarcinoma (using biopsy from the initial diagnostic endoscopy)
Suitable for potentially curative surgical resection
No contraindications for treatment with pembrolizumab
Have impending bowel obstruction or other indications for acute surgical intervention
Have had concurrent immunotherapy in the 3 months before the start of the study therapy.
Have acute or chronic hepatitis B or hepatitis C infection
Known HIV disease
Single group
BT-001 followed by Pembrolizumab (PD-1 Blocking Antibody)
DrugTwo doses of BT-001 delivered by intra-tumoural injection followed by one systemic dose of pembrolizumab
Overall incidence of adverse events (AEs)
Evaluated according to National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Overall incidence of serious adverse events (SAEs)
Evaluated according to National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Overall incidence of dose limiting toxicities (DLTs)
Incidence of dose-limiting toxicities
Clinical efficacy of BT-001 when delivered by endoscopic/transrectal ultrasound guided intra-tumoural injection in combination with a single systemic dose of pembrolizumab in patients with primary, localised, rectal cancer with proficient mismatch repair
Defined as the proportion of patients achieving a complete or major pathological response.
Effects of the study treatment on long-term oncological outcomes
Percentage of patients developing local recurrence and/or distant metastases at 1-, 3- and 5-years' follow-up
Determine the effects of the study treatment on patient's quality of life
Assessed using the EORTC QLQ (European Organisation for Research and Treatment of Cancer Qulaity of Life Questionaire) CR29 questionnaire at baseline, 1 month after surgery, and 1-, 3- and 5-years' follow-up. Higher score means a poorer outcome. Minimum score 29, maximum score 116.
Determine the effects of the study treatment on patient's quality of life
Assessed using the EQ (Euroqol) 5D questionnaire at baseline, 1 month after surgery, and 1-, 3- and 5-years' follow-up. Higher scores mean a better outcome. Minimum score 0, maximum score 100
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Henry Smith
Lead sponsor
Bispebjerg Hospital
Sponsor institution
Transgene
Collaborator
Rigshospitalet, Denmark
Collaborator
