huCART-meso + VCN-01 in Pancreatic and Ovarian Cancer

NCT05057715 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: UPCC# 03821, IND #27590
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase I study evaluating the safety and feasibility of lentiviral transduced huCART-meso cells when given in combination with VCN-01 in a 3+3 dose (de)escalation design.

Conditions

Pancreatic Cancer; Serous Ovarian Cancer

Outcome Measures

Primary Outcomes (2)

• Type, frequency, severity, and attribution of AEs/SAEs as assessed by CTCAE v 5.0

  2 years

• Occurrence of dose-limiting toxicities.

  2 years

Secondary Outcomes (6)

• Proportion of subjects enrolled who receive one or both of the intended study infusions

  2 years

• Overall Response Rate (ORR)

  15 years

• Best Overall Response (BOR)

  15 years

• Duration of Response (DOR)

  15 years

• Progression Free Survival (PFS)

  15 years

Study Arms (3)

Cohort 1

ACTIVE COMPARATOR

Single dose of 3.3x10(12) vp of VCN-01 on Day 0, followed by a single dose of 5x10(7) of huCART-meso cells on Day 14.

Biological: VCN-01; Biological: huCART-meso Cells

Cohort 2

ACTIVE COMPARATOR

Single dose of 1x10(13) vp of VCN-01 on Day 0, followed by a single dose of 5x10(7) of huCART-meso cells on Day 14.

Biological: VCN-01; Biological: huCART-meso Cells

Cohort -1

ACTIVE COMPARATOR

In the event that 2 DLTs occur in Cohort 1, then enrollment in Cohort 1 will be stopped and Cohort -1 will be opened for evaluation. Enrolled subjects will receive a single dose of huCART-meso cells on Day 0 followed by a single dose of 3.3x10(12) vp of VCN-01 on Day 14.

Biological: VCN-01; Biological: huCART-meso Cells

Interventions

VCN-01 BIOLOGICAL

Intravenous administration of VCN-01

Cohort -1; Cohort 1; Cohort 2

huCART-meso Cells BIOLOGICAL

Intravenous administration of huCART-meso cells

Cohort -1; Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with one of the following diagnoses:
• Histologically confirmed unresectable or metastatic pancreatic adenocarcinoma; OR
• Persistent or recurrent serous epithelial ovarian cancer
• Progression or intolerance to at least one prior standard of care chemotherapy for advanced stage disease.
• Subjects must have measurable disease as defined by RECIST 1.1 criteria.
• Patients ≥ 18 years of age.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• Adequate organ and bone marrow function defined as:
• Hemoglobin ≥ 9 g/dL
• Platelets ≥ 75,000/µl
• PT/INR and PTT ≤ 1.5 x ULN
• Bilirubin ≤ 2.0 x ULN
• Creatinine ≤ 1.5 x ULN
• ALT/AST ≤ 5 x ULN (subjects with liver metastases) or ALT/AST ≤ 2.5 x ULN (subjects without liver metastases)
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygen \> 92% on room air

You may not qualify if:

• Patients with known CNS metastases
• Active invasive cancer other than the one of the two cancers targeted by this study. Patients with active non-invasive cancers (such as non-melanoma skin cancer, superficial cervical and bladder and prostate cancer with PSA level \< 1.0) are not excluded.
• Active hepatitis B or hepatitis C infection.
• Chronic hepatitis C with a FibroScan score equivalent to fibrosis stage 2 (F2) or greater.
• Patients with known cirrhosis.
• Patients with ongoing or active infection.
• Patients with a known history of Li Fraumeni syndrome or retinoblastoma protein pathway germinal deficiency.
• Active autoimmune disease requiring systemic immunosuppressive treatment equivalent to ≥ 10 mg of prednisone. Patients with autoimmune neurologic diseases (such as MS) will be excluded.
• Planned concurrent treatment with systemic high dose corticosteroids. Patients may be on a stable low dose of steroids (≤ 10mg equivalent of prednisone). Use of inhaled steroids is allowable.
• Patients requiring supplemental oxygen therapy.
• History of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
• Any clinically significant pericardial effusion, Class II-IV cardiovascular disability according to the New York Heart Association Classification (see Appendix 4) or other cardiovascular condition that would preclude assessment of mesothelin induced pericarditis or that may worsen as a result of toxicities expected for this study. This determination will be made by a cardiologist if cardiac issues are suspected.
• Pregnant or breastfeeding women.
• Treatment with a PD-1 or PD-L1 inhibitor, including but not limited to nivolumab, pembrolizumab, atezolizumab, and/or durvalumab, within 2 months prior to eligibility confirmation by a physician-investigator.
• Patients with significant lung disease as follows:

Sponsors & Collaborators

• University of Pennsylvania: lead
• Theriva Biologics: collaborator

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Pancreatic Neoplasms

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases

Study Officials

• Janos L. Tanyi, MD, PhD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 3+3

Study Record Dates

• First Submitted: September 15, 2021
• First Posted: September 27, 2021
• Study Start: March 2, 2022
• Primary Completion (Estimated): September 1, 2038
• Study Completion (Estimated): September 1, 2038
• Last Updated: June 25, 2025

Record last verified: 2025-06

Locations

US (1)