Table 1.

Summary of our clinical trials of GPC3‐derived peptide vaccine

Trial	UMIN	Key inclusion criteria	Primary endpoint	Results
Phase I clinical study of GPC3 peptide vaccine in patients with advanced HCC	000001395	Advanced HCC patient	Adverse effects of GPC3 vaccination GPC3‐specific immune responses to GPC3 vaccination	GPC3 vaccination was well‐tolerated The vaccine induced a GPC3‐specific CTL response in 91% patients (30/33)
Clinical study evaluating immunological efficacy of GPC3 peptide vaccine in patients with advanced HCC	000005093	Advanced HCC patient	Increase of frequency of GPC3‐peptide specific CD8 positive T lymphocytes in the blood and into the tumor	After the vaccination, the number of GPC3 peptide‐specific CTLs in PBMC was found to have increased in 9 of 11 patients and tumor biopsy specimens after the vaccination ware obtained 3 patients, in which they found to infiltrate into the tumor
A Phase II study of GPC3 peptide vaccine as adjuvant treatment for HCC after surgical resection or Ragiofrequency ablation (RFA)	000002614	Diagnosed as initial HCC Subjects who undergone potentially curative surgical resection or RFA for treatment of HCC	The 1‐ and 2‐y recurrence rate	The 1‐ and 2‐y recurrence rates were 24.4% and 53.7%, respectively. The primary endpoint was not reached
Phase II study of GPC3 peptide vaccine as treatment for OCCC	000003696	Advanced OCCC patient	DCR at 6 mo	DCR at 6 mo was 9.4% (3/32)
A Phase I study of GPC3 peptide vaccine for pediatric patients with refractory tumors	000006357	Patients in refractory, recurrent, or progressive status Patients in remission without chance of cure Patients in PR or SD	Incidence of DLT	No DLT or dose‐specific adverse events were observed

GPC3, glypican‐3; HCC, Hepatocellular carcinoma; OCCC, ovarian clearcell carcinoma; PR, partial response; SD, stable disease; DCR, disease control rate; DLT, dose limiting toxicity; CTL, cytotoxic T lymphocyte.