CAR T Therapy With GCAR1 for Relapsed Alveolar Soft Part Sarcoma

An Open Label Individual Patient Dose Escalation Study Investigating the Safety and Efficacy of Retreatment With GCAR1 in a Patient With Multiply Relapsed Alveolar Soft Part Sarcoma

A single patient study to determine whether GCAR1 is safe and effective for re-treatment of alveolar soft part sarcoma (ASPS) with GPNMB surface expression that has relapsed and is not responding to usual treatment.

CLIC-YYC-GPNMB-04 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-04 has refractory, progressive metastatic alveolar soft part sarcoma (ASPS). There are no standard therapies for relapsed ASPS known to provide potential for cure, and there are no clinical trials available in Canada for consideration. We propose to re-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). The patient was previously treated with GCAR1 under CLIC-YYC-GPNMB-01 (c#276646).

Inclusion Criteria:

• The patient must provide informed consent
• Adequate organ function, defined as creatinine clearance >30 ml/min and LVEF >45%

Exclusion Criteria:

• Any active uncontrolled infection
• Pregnancy or nursing
• Anti-cancer therapy within 21 calendar days prior to the first dose of lymphodepleting chemotherapy. If subject has received anti-cancer therapy, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy as per the discretion of the Qualified Investigator/Co-Investigator