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

NCT07104682 | Not Yet Recruiting Early Phase 1

• 1 other identifier: HREBA.CC-25-0218
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Alveolar Soft Part Sarcoma (ASPS); Sarcoma

Outcome Measures

Primary Outcomes (1)

• Treatment response

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

  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.

Study Arms (1)

GCAR1

EXPERIMENTAL

The patient may receive two separate intravenous infusions of cryopreserved, autologous GCAR1, each containing a total of 5.0E6 CAR+ T cells/kg patient body weight. Both infusions will be preceded by standard lymphodepleting chemotherapy (Fludarabine 40 mg/m2 x 3 days, cyclophosphamide 600 mg/m2 x 2 days). The patient will only be eligible to receive a second dose if they had a partial response (PR), stable, or progressive disease (SD/PD) by RECIST 1.1 criteria after receiving Dose 1. A second infusion should not be administered if the patient achieves and sustains a complete response on disease assessment. Dose 2 will be administered at physician discretion and must be given at minimum 60 days and a maximum of 730 days post administration of Dose 1. For the patient to proceed with a second infusion, any toxicity that does not meet the criteria of a Dose Limiting Toxicity must be assessed for clinical significance, and taken into consideration by the PI before proceeding.

Biological: GCAR1

Interventions

GCAR1 BIOLOGICAL

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

GCAR1

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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%.

You may not qualify if:

• Any active uncontrolled infection
• Any anti-cancer therapy within 21 calendar days prior to the first dose of lymphodepleting chemotherapy

Sponsors & Collaborators

• University of Calgary: lead

Study Sites (1)

University of Calgary

Calgary, Canada

Location

MeSH Terms

Conditions

Sarcoma, Alveolar Soft Part; Sarcoma

Condition Hierarchy (Ancestors)

Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Single Group Assignment

Study Record Dates

• First Submitted: July 29, 2025
• First Posted: August 5, 2025
• Study Start: August 8, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: August 5, 2025

Record last verified: 2025-07

Locations

CA (1)