A Phase 1 Open-Label, Multi-Center First in Human Study of TnMUC1-Targeted Genetically-Modified Chimeric Antigen Receptor T Cells in Patients with Advanced TnMUC1-Positive Solid Tumors and Multiple Myeloma
Multi-center, open-label, first in human Phase 1 study of the safety, tolerability, feasibility, and preliminary efficacy of the administration of genetically modified autologous T cells (CART-TnMUC1 cells) engineered to express a chimeric antigen receptor (CAR) capable of recognizing the tumor antigen, TnMUC1 and activating the T cell (CART- TnMUC1 cells).
Primary Objectives: Phase 1: Identify the RP2D for further study of CART-TnMUC1 cells that can be administered safely in patients with either TnMUC1 positive refractory solid tumors (Arm 1) or TnMUC1 positive relapsed/refractory multiple myeloma (Arm 2) in combination with the lymphodepletion (LD) chemotherapy regimen. Phase 1a expansion: Estimate the Objective Response Rate (ORR) of patients with TnMUC1+ platinum resistant ovarian cancer treated with CART-TnMUC1 cells and LD chemotherapy regimen. Secondary Objectives (Phase 1 and Phase 1a expansion): - Assess the safety, tolerability and feasibility of CART-TnMUC1+ refractory solid tumors and relapsed/refractory TnMUC1+ multiple myeloma. -Determine the preliminary anti-tumor efficacy of CART-TnMUC1+ refractory solid tumors and relapsed/refractory multiple myeloma with the endpoints of ORR, OS, Duration of Response (DOR), disease control rate (DCR), Time to Response (TTR), progression free survival (PFS), and minimal residual disease status (MRD). -Correlation of the expansion and persistence of CART-TnMUC1 with efficacy parameters. -Correlation of peripheral expansion and persistence of CART-TnMUC1 cells with related efficacy and safety parameters. Exploratory Objectives (Phase 1 and Phase 1a expansion): -Characterize the clinical pharmacology of the CART-TnMUC1 cells with respect to their expansion, persistence, trafficking, effector status and function (eg. cytokine release) within the periphery and the tumor microenvironment. -Evaluate the pattern of tumor markers (e.g., tumor microenvironment [TME] markers of inflammation and immunosuppression) and soluble disease markers (e.g., CA-125, soluble MUC1, CA19.9) with relation to both safety and efficacy endpoints. -Evaluate the pattern of safety and efficacy in patients pre-treated with prior CART therapy. -Evaluate tumor antigen immune escape mechanisms in tumor biopsies and peripheral blood. -Evaluate the development of anti-CAR immune responses. Secondary Long-term Follow-up Objectives (Phase 1 and Phase 1a expansion): -Evaluate the incidence of new malignancies and pre-malignant conditions in all patients. -Evaluate the de novo incidence of or exacerbation of pre-existing neurologic disorders in all patients. -Evaluate the de novo incidence of or exacerbation of prior rheumatologic or other autoimmune disorders. -Evaluate the de novo incidence of or exacerbation of prior hematologic disorders. -Evaluate vector persistence by assessing the detectable TnMUC1 CAR transgene levels in peripheral blood by q-PCR at pre-specified post CART-TnMUC1 infusion time points. -Evaluate patients for the presence of detectable RCL by VSV-G.
Chemotherapy (NOS); Immunotherapy
CART-TnMUC1 (); cyclophosphamide (); cytoxan (cyclophosphamide); fludarabine (Fludarabine phosphate)
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