CAR T Therapy With GCAR1 for 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Sarcoma Alveolar Soft Part Sarcoma (ASPS) Neoplasms Neoplasms by Histologic Type Neoplasms, Connective and Soft Tissue Neoplasms, Muscle Tissue Sarcoma, Alveolar Soft Part

Sex eligibility

Female

Study type

Interventional

Phase

Early Phase 1

Primary location

Arthur J.E. Child Comprehensive Cancer Centre

Calgary, Alberta, T2N 5G2, Canada

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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, and the CD137 (4-1BB) and CD3 zeta chain intracellular signaling domains. Following 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 immunosurveillance3.

Primary outcomes

Treatment Response

Time frame: Diagnostic imaging (CT and/or MRI) will be performed at baseline (pre-treatment) and then subsequently at 4-6 weeks after GCAR1 infusion, and at 3 months, 6 months, 9 months and 1 year 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.

Response Classification of Target Lesions, CNS lesions and Non-Target Lesions
- Complete response (CR):
- Partial response (PR):
- Progressive disease (PD):
- Stable disease (SD):

Sponsors and collaborators

Lead sponsor

University of Calgary

Other

Collaborators

Alberta Health Services, Calgary
Alberta Precision Laboratories

Registry information

Official study title

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

Acronym: SPS-Q2

Important dates

Study start: 2025
Primary completion: 2030
Study completion: 2030
First posted: Feb 7, 2025
Registry last updated: Apr 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Not Yet Recruiting