Table 1.
Clinical trials of GPC3-targeted antibody therapy and vaccine therapy.
| Study Title | Phase | Trial No. | Conditions | Outcomes | Reference |
| Molecular targeting therapy | |||||
| A phase I study of GC33 in advanced or metastatic liver cancer (hepatocellular carcinoma) | I | NCT 00746317 |
Advanced or metastatic HCC | GC33 was well tolerated. GPC3 expression in HCC may be associated with the clinical benefit to GC33. |
Zhu et al. 2013 [63] |
| Phase I study of GC33 in patients with advanced HCC | I | JapicCTI 101255 |
Japanese patients with advanced HCC | GC33 was well tolerated. The correlation between antitumor activity and GPC3 expression was not clear. |
Ikeda et al. 2014 [64] |
| A study of RO5137382 (GC33) in patients with advanced or metastatic HCC | II | NCT 01507168 |
Patients with advanced HCC who had failed prior systemic therapy | Codrituzumab did not show any clinical benefit. A high dose of codrituzumab or a high GPC3 level or its mediator CD16 may improve outcome. |
Abou-Alfa et al. 2016 [65] |
| Vaccines therapy | |||||
| Phase I clinical study of glypican-3 peptide vaccine in patients with advanced HCC | I | UMIN 000001395 |
Advanced HCC patients | GPC3 vaccination was well tolerated. Peptide-specific CTL frequency may be a predictive marker of OS. |
Sawada et al. 2012 [71] |
| A phase II study of GPC3 peptide vaccine as adjuvant treatment for HCC after surgical resection or radiofrequency ablation (RFS) | II | UMIN 000002614 |
Patients with initial HCC who had undergone surgery or radiofrequency ablation | GPC3 vaccination did not have longer RFS or OS. Vaccination in patients with GPC3-positive tumors improved 1-y recurrence rates. |
Sawada et al. 2016 [72] |
| Ongoing GPC3-Targeted Antibody Therapy Trials | Phase | Trial No. | Conditions | Status | Country |
| A study of ERY974 in patient with advanced solid tumors | I | NCT 02748837 |
GPC3-positive advanced solid tumors | Active, not recruiting | United States |
| A phase I study of codrituzumab, in combination with atezolizumab in patients with HCC | I | JapicCTI 163325 |
Locally advanced or metastatic HCC in which GPC3 is expressed by IHC | Active, not recruiting | Japan, Taiwan |
CTL: cytotoxic T lymphocyte; OS: overall survival; RFS: recurrence-free survival; IHC: immunohistochemistry.