A Study to Determine the Safety and Effectiveness of the Investigational Cellular Therapy GCAR1 in a Patient With Alveolar Soft Part Sarcoma

Condition(s) studied

Investigational drug(s) / intervention(s)

GCAR1: GCAR1 is a patient-specific cell therapy product containing a mixture of autologous lymphocytes transduced with a lentiviral vector encoding a chimeric antigen receptor (CAR) targeting GPNMB. The CAR comprises a single-chain variable fragment (scFv) binding domain derived from a fully human GPNMB-specific monoclonal antibody (CDX-011), a CD8 hinge and transmembrane domain, a myc sequence for product identification, and the CD137 (4-1BB) and CD3 zeta chain intracellular signaling domains. After infusion, the autologous GPNMB CAR T-cells expressing the genetically engineered anti-GPNMB CAR enable the specific targeting of GPNMB-expressing cells. Upon binding to GPNMB-expressing cells, the CAR transmits T cell activation signals that promote the elimination of target cells through CAR T cell degranulation and the release of cytotoxic molecules. The cellular signal also facilitates CAR T cell proliferation and persistence that may enable prolonged disease control through immunosurveillance

Study summary

A single patient study to determine whether GCAR1 is safe and effective for refractory, progressive metastatic alveolar soft part sarcoma (ASPS).

Eligibility

Primary outcome measure(s)

• Treatment response — Diagnostic imaging (CT and/or MRI) will be performed at baseline (pre-treatment) and then subsequently at day 46 and day 91 after GCAR1 infusion, and at 6 months, 9 months and 12 months after last infusion to evaluate response to therapy.
  The overall response assessment considers the response of the target and non-target lesions and development of new lesions.

Trial sites (1)

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