A Study to Determine the Safety and Effectiveness of the Investigational Cellular Therapy GCAR1 in a Patient With Alveolar Soft Part Sarcoma (CLIC-YYC-GPNMB)

University of Calgary

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Sarcoma; Alveolar Soft Part Sarcoma (ASPS)

Treatments: Biological: GCAR1

Study type: Interventional

Funder types: Other

Identifiers

NCT07104682

HREBA.CC-25-0218

Details and patient eligibility

About

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

Full description

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).

Enrollment: 1 estimated patient

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

GCAR1

Experimental group

Description:

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.

Treatment:

Biological: GCAR1