This is an open-label phase 1 study to assess the safety and feasibility of autologous T cells expressing a single-chain scFv targeting GFRα4 with tandem TCR/CD3ζ and 4-1BB (TCRζ/4-1BB) co-stimulatory domains (referred to as "CART-GFRa4 cells") in patients with incurable medullary thyroid cancer (MTC).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Enrollment
18
Intravenous infusion of lentiviral transduced autologous T cells that have been engineered to express an extracellular single chain antibody (scFv) with specificity towards GFRa4 with fludarabine and cyclophosphamide.
Lymphodepletion
Lymphodepletion
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v5.0.
Time frame: 15 years
Percentage of manufacturing products that meet release criteria.
Time frame: 3 months
Number of subjects who have a response (ORR)
Time frame: 12 months
Best Overall Response (BOR)
Time frame: 12 months
Duration of Response (DOR)
Time frame: 12 months
Overall survival (OS)
Time frame: 12 months
Progression-free survival (PFS)
Time frame: 12 months
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