RR001, a cell-based gene therapy administered following chemotherapy cycles for the treatment of patients with locally advanced pancreatic cancer. Phase I /IIa clinical trial (open label and non-randomized) to test the effects (safety \& efficacy) of increasing doses of RR001
This study is intended for people diagnosed with locally advanced pancreatic ductal adenocarcinoma. Currently, surgical resection is the only treatment that offers a chance to cure this type of tumor, but only a few percentage of locally advanced pancreatic adenocarcinoma patients is able to benefit from this approach. Thus, research is aimed at identifying new treatment strategies based on innovative approaches that can increase the number of people who can access surgery as radically as possible. The SNIPER study aims to evaluate for the first time in humans the applicability and safety of a new drug (called RR001) based on autologous cells, that are, cells taken from the same subject who receives them. These cells are derived from adipose tissue and are genetically modified to deliver a potent anti-cancer death ligand normally produced by the body with the role to kill tumoral cells.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)
Azienda Ospedaliero-Universitaria (AOU) Policlinico di Modena
Modena, Italy, Italy
RECRUITINGEvaluation of Safety in terms of monitoring of systemic tolerance, Adverse Events (AEs) and severe AEs (SAEs) assess by CTCAE (Version 5.0)
Time frame: From the signing of the informed consent onwards and during every visit till the End of Study (EOS) (90±7 days after RR001 delivery) for a total of 26 weeks
Evaluation of feasibility through the assessment of the production capacity of the Cell Factory participating in the study of an adequate quantity of autologous cellular product lots sufficient to treat 80% of the patients recruited
Time frame: From Day 1 till RR001 delivery (46-57 days)
Evaluation of the Efficacy of the Drug Product: Overall response rates (ORR)
Overall response rates (ORR) per RECIST 1.1 criteria till End Of Treatment (EOT)
Time frame: 21 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product: percentage of pathological resection
Percentage of patient undergoing surgery and percentage of pathological resection
Time frame: At Day 24 post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Time to progression (TTP) till end of study
Time frame: Day 90± 7 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Progression free survival (PFS) till end of study
Time frame: Day 90± 7 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Overall survival (OS) till end of study
Time frame: Day 90± 7 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Tumoral markers evaluation
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Tumoral markers evaluation CA 19.9, CEA till end of study
Time frame: Day 90±7 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Quality of Life (QoL) Questionnaire of European Organization for Research and Treatment of Cancer (EORTC) - EORTC QLQ-C30
Questionnaire on Quality of Life (QoL) collected till the end of the study. The EORTC Quality of Life Group (QLG) Core Questionnaire (EORTC QLQ-C30) is a 30 item instrument meant to assess some of the different aspects that define the QoL of cancer patients. The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems.
Time frame: Day 90± 7 days post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Quality of Life (QoL) Questionnaire of European Organization for Research and Treatment of Cancer (EORTC) - EORTC QLQ-PAN26
Questionnaire on Quality of Life collected till the end of the study EORTC QLQ-PAN26 (Pancreatic Cancer Module). This questionnaire is administered on the same module of EORTC QLQ-C30 Questionnaire. The module comprises 26 questions assessing pain, dietary changes, jaundice, altered bowel habit, emotional problems related to pancreatic cancer, and other symptoms (cachexia, indigestion, flatulence, dry mouth, taste changes). All of the scales and single-item measures range in score from 0 to 100, where zero corresponds to the lowest possible score and 100 to the highest possible score.
Time frame: Day 90± 7 days post RR001 delivery