CAR-GPC3 T Cells in Patients With Refractory Hepatocellular Carcinoma
- Conditions
- Hepatocellular Carcinoma
- Interventions
- Genetic: CAR-GPC3 T cells
- Registration Number
- NCT03146234
- Lead Sponsor
- RenJi Hospital
- Brief Summary
This study is designed to determine the safety and efficacy of CAR-GPC3 T cells in patients with relapsed or refractory hepatocellular carcinoma. Single or multiple doses of GPC3-targeted CAR T cells will be given to subjects with unmet medical needs for which there are no effective therapies known at this time.
- Detailed Description
A single arm, open-label pilot study is designed to determine the safety, tolerability and engraftment potential of CAR-GPC3 T cells in patients with GPC3-positive hepatocellular carcinoma.
Primary objectives:
Determine the safety, tolerability and cytokinetics of the autologous T cells transduced with the anti- GPC3 lentiviral vector in patients with hepatocellular carcinoma.
Secondary objectives:
Make a preliminary evaluation on the efficacy of CAR-GPC3 T cells in patients with hepatocellular carcinoma by the following parameters:
Objective response rate (ORR); Disease Control Rate (DCR); Time of tumor progression (TTP); Overall survival (OS).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 7
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CAR-GPC3 T cells CAR-GPC3 T cells Autologous T Cells with a GPC3-redirected Chimeric Antigen Receptor. Route of administration: Intravenous injection. Lymphodepletion conditioning regimen: A combination of fludarabine and cyclophosphamide will be administered at Day -6 to Day -3 prior to CAR-GPC3 T cells infusion.
- Primary Outcome Measures
Name Time Method Safety and tolerance 24 weeks Study related adverse events are defined as signs above CTCAE Grade 3, laboratory toxicities and clinical events occurred at any time from the first day of infusion to week 24 that are "possibly", "likely", or "definitely" related to the study, including infusion related toxicity and CAR-GPC3 T cells related toxicity. Include but not limited to:
Fever; Chills; Nausea, vomiting and other gastrointestinal symptoms; Fatigue; Hypotension; Respiratory distress; Tumor lysis syndrome; Cytokine release syndrome; Neutropenia, thrombocytopenia; Liver and kidney dysfunction; Other toxicities.
- Secondary Outcome Measures
Name Time Method Engraftment 2 years Duration of in vivo survival of CAR-GPC3 T cells is defined as "engraftment". The primary engraftment endpoint is the number of DNA vector copies per mL blood of CAR-GPC3 T cells at regular intervals through week 4 following the initial infusion. Q-PCR for CAR-GPC3 vector sequences will be performed until any 2 sequential tests are negative, documented as engraftment and persistence of CAR-GPC3 T cells.
Trial Locations
- Locations (1)
Renji Hospital, Shanghai Jiaotong University School of Medicine
🇨🇳Shanghai, Shanghai, China