Phase I/IIa Study: RR001 Administered Following Chemotherapy Cycles For Patients With Locally Advanced Pancreatic Carcinoma

Study Overview

Phase I/IIa Study: RR001 Administered Following Chemotherapy Cycles for Patients With Locally Advanced Pancreatic Carcinoma

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.

Locally Advanced Pancreatic Adenocarcinoma

DRUG: RR001

SNIPER
2024-516019-26-00 (CTIS Identifier) (CTIS: )

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates	Results Reporting Dates	Study Record Updates
First Submitted 2025-01-28	Results First Submitted N/A	Last Update Submitted that met QC Criteria 2025-07-01
First Submitted that Met QC Criteria 2025-03-04	Results First Submitted that Met QC Criteria N/A	Last Update Posted (ACTUAL) 2025-07-02
First Posted (ACTUAL) 2025-03-06	Results First Posted N/A	Last Verified 2025-07

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

Primary Purpose:
Treatment

Allocation:
Non Randomized

Interventional Model:
Sequential

Masking:
None

Arms and Interventions

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: RR001 Dose Level 1 Treatment consists of two cycles of Nab-PTX 125 mg/mq followed by GEM 1000 mg/mq administered on days 1, 8, and 15, every 28 days. At the end of the 2 cycles patients will undergo to an intra-tumoral administration of RR001 at Dose Level (DL) 1: 80 x 10^6	DRUG: RR001 RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)
EXPERIMENTAL: RR001 Dose Level 2 Treatment consists of two cycles of Nab-PTX 125 mg/mq followed by GEM 1000 mg/mq administered on days 1, 8, and 15, every 28 days. At the end of the 2 cycles patients will undergo to an intra-tumoral administration of RR001 at Dose Level (DL) 2: 160 x 10^	DRUG: RR001 RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)
EXPERIMENTAL: RR001 Dose Level 3 Treatment consists of two cycles of Nab-PTX 125 mg/mq followed by GEM 1000 mg/mq administered on days 1, 8, and 15, every 28 days. At the end of the 2 cycles patients will undergo to an intra-tumoral administration of RR001 at Dose Level (DL) 3: 240 x 10^	DRUG: RR001 RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)

Participant Group/Arm

Intervention/Treatment

EXPERIMENTAL: RR001 Dose Level 1

Treatment consists of two cycles of Nab-PTX 125 mg/mq followed by GEM 1000 mg/mq administered on days 1, 8, and 15, every 28 days. At the end of the 2 cycles patients will undergo to an intra-tumoral administration of RR001 at Dose Level (DL) 1: 80 x 10^6

DRUG: RR001

RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)

EXPERIMENTAL: RR001 Dose Level 2

DRUG: RR001

RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)

EXPERIMENTAL: RR001 Dose Level 3

DRUG: RR001

RR001, a cell-based gene therapy administered following administration of chemotherapy (Nab-PTX 125 mg/mq and GEM 1000 mg/mq)

Primary Outcome Measures	Measure Description	Time Frame
Evaluation of Safety in terms of monitoring of systemic tolerance, Adverse Events (AEs) and severe AEs (SAEs) assess by CTCAE (Version 5.0)		From the signing of the informed consent onwards and during every visit till the End of treatment (EOT) (21 days after RR001 delivery) for a total duration up to 16 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 90% of the patients recruited		From Day 1 till RR001 delivery (46-57 days)

Secondary Outcome Measures	Measure Description	Time Frame
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)	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	At Day 24 post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Time to progression (TTP) till end of study		Day 90 post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Progression free survival (PFS) till end of study		Day 90 post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Overall survival (OS) till end of study		Day 90 post RR001 delivery
Evaluation of the Efficacy of the Drug Product : Tumoral markers evaluation	Tumoral markers evaluation CA 19.9, CEA till end of study	Day 90 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.	Day 90 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.	Day 90 post RR001 delivery

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: Annalisa Fontana

Phone Number: IT + 39 0594223808/0594225165

Email: fontana.annalisa@aou.mo.it

Study Contact Backup

Name: Andrea Spallanzani

Phone Number: IT +39 0594223310/0594225646

Email: spallanzani.andrea@aou.mo.it

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person’s general health condition or prior treatments.

Ages Eligible for Study:
ALL

Sexes Eligible for Study:
18 Years

Accepts Healthy Volunteers:

Patients with confirmed pancreatic ductal adenocarcinoma classified as locally advanced pancreatic adenocarcinoma (LPAC)
Patients with no evidence of peritoneal or hematogenous metastasis
Patients classified as non-resectable locally advanced pancreatic carcinoma (LAPC) based on imaging (TC and NMR), on multidisciplinary staff evaluation by at least an oncologist, radiologist and a qualified digestive surgeon and accounting for AJCC/UICC TNM and clinical staging
Measurable tumor according RECIST criteria v 1.1
Low tumor burden with at least one lesion equal/less than 3,5 cm that is suitable for US guided injection (and needle biopsy).
Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
Patients must be eligible for chemotherapy treatment (based on standard of care) treatment
Patient older than 18 years of age
Adequate hepatic and kidney function/Safe hematologic profile
Negative serum pregnancy test for females of childbearing potential
Willingness and ability to comply with the scheduled visits, treatment plan, imaging procedures, laboratory tests and other study procedures, including lipoaspirate collection (liposuction)

Patient with pancreatic cystic tumor or pancreatic pseudocyst
Patient with pancreatic tumor different from adenocarcinoma (endocrine, metastases)
Patients with unknown stage or recurrent pancreatic cancer
Patients with immunosuppression or susceptibility to viral infection
Patients with HIV, hepatitis B, hepatitis C, HTLV-I/II, Treponema Pallidum infections
Patients with liver cirrhosis or other documented liver diseases
Patient contraindication to use chemotherapy treatments
Previous of radiotherapy and chemotherapy for PDAC
Previous hematopoietic stem cell or organ transplantation
Irreversible cardiac arrhythmias requiring permanent medication
Heart insufficiency (> grade II, New York Heart Association NYHA criteria)
History within the last year of acute or subacute coronary syndromes including myocardial infarction, unstable or severe stable angina pectoris
Uncontrolled hypertension
Other malignancies within the past 2 years (not including basal cell carcinoma of the skin, prostate cancer or in situ cervix carcinoma, in situ melanoma).
Active autoimmune disease
Use of any investigational agents within 21 days from the administration of study treatment
Patient has had major open surgery prior to the administration of study treatment
Uncontrolled intercurrent illness including but not limited to psychiatric illness/social situations that in the opinion of the Investigator would compromise compliance of study requirements or put the patient at unacceptable risk

Collaborators and Investigators

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