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

Early Phase 1Not Yet RecruitingNCT07104682

University of Calgary1 enrolled

Overview

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

CLIC-YYC-GPNMB-05 is an Open Label Individual Patient (OLIP)/Single Patient Study (SPS) developed according to the Health Canada template and guidelines released in 2019 for studies to access therapies not otherwise available to patients, in the situation where there are no options of treatment or cure remaining. The patient under consideration for CLIC-YYC-GPNMB-05 has refractory, progressive metastatic alveolar soft part sarcoma (ASPS). There are no standard therapies for metastatic ASPS known to provide potential for cure, and there are no clinical trials available in Canada for consideration. We propose to treat the patient with GCAR1, a patient-specific cell therapy product containing a mixture of autologous lymphocytes transduced with a lentiviral vector containing a chimeric antigen receptor (CAR) that enables the specific targeting towards tumor cells expressing the cell surface protein glycoprotein non-metastatic B (GPNMB).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Alveolar Soft Part Sarcoma (ASPS)Sarcoma

Interventions

GCAR1BIOLOGICAL

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

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: * The patient must have relapsed ASPS that is in the opinion of the treating physician not resectable, or that resection would be associated with significant morbidity. * The patient must provide informed consent. * The only other eligibility criteria is adequate organ function, defined as creatinine clearance \>30 ml/min and LVEF \>45%. Exclusion Criteria: * Any active uncontrolled infection * Any anti-cancer therapy within 21 calendar days prior to the first dose of lymphodepleting chemotherapy

Locations (1)

University of Calgary

Calgary, Canada

Outcomes

Primary Outcomes

Treatment response

The overall response assessment considers the response of the target and non-target lesions and development of new lesions.

Time frame: 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.

Data from ClinicalTrials.gov

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