NCT05033522

Immunotherapy for Advanced Liver Cancer

Phase II/III Randomized, Controlled Clinical Study of AlloStim(R) vs Physician's Choice in Asian Subjects With Advanced Hepatocellular Carcinoma

• Status: Suspended
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: Mirror Biologics, Inc. · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a randomized, controlled multi-site, multi-national clinical trial conducted in Thailand and Malaysia for Asian adults (males or females), 18 years of age and older presenting with advanced HCC (BCLC stage C) including subjects with macrovascular involvement and/or extrahepatic spread (not eligible for TACE, surgery or locoregional treatment) with Child-Pugh stage A or B liver function. 150 subjects will be randomized 2:1 to AlloStim® immunotherapy vs Physician's Choice of Sorafenib, Lenvatinib or FOLFOX4.

Detailed description

A multi-national, randomized, controlled trial (RCT) with multiple sites selected in Asia (Malaysia and Thailand). Subjects with no prior treatments for BCLC stage C disease and presenting with Child-Pugh class A or B liver reserve to be randomized 2:1 to AlloStim® vs. Physician's Choice (PC). PC to be declared prior to randomization. PC allowed to be either sorafenib, levantinib or FOLFOX4. The world incidence of hepatocellular carcinoma (HCC) is highest in East and South East Asia, with nearly half of the all HCC cases and deaths globally occurring in China. In Asian countries, the main treatment options for early or intermediate HCC (BCLC class A and B) include surgical resection, ablation (e.g., RFA, ETOH, cryoablation), transarterial chemoembolization (TACE), radiation or systemic chemotherapy depending on liver function status. However, in the Asian-Pacific region it is estimated that up to 80% of patients present with unresectable, advanced HCC (BCLC Stage C) that are not eligible for locoregional therapy, surgery or TACE due to tumor size and/or vascular involvement. For these patients, the standard of care for over a decade has been sorafenib (Bayer, A.G.), a oral kinase inhibitor based on the results of a RCT (SHARP study) of 602 patients randomized to sorafenib vs. placebo. Median overall survival (OS) was 10.7 months for sorafenib and 7.9 months for placebo (p\<0.05). The SHARP study included a Western population. A separate study in Asian patients (226 patients from China, South Korea and Taiwan) comparing sorafenib to placebo (Sorafenib-AP study) demonstrated a OS of 6.5 months for sorafenib compared to 4.2 months for placebo (p\<0.05). The placebo OS difference between Asian and Western patients (4.2mo vs 7.9 mo) suggests a difference in the disease characteristics in the Asian population. One significant difference is that the Asian population has an increased prevalence of HBV compared to Western population which may contribute to the increased incidence of HCC and worse OS outcomes observed in Asian patients compared to Western patients. In Thailand and Malaysia sorafenib is not available to a majority of the population presenting with advanced HCC, both due to cost and toxicity profile. This study is designed to evaluate whether AlloStim ® immunotherapy will provide a survival benefit to this population with an improved quality of life compared to approved first line therapy.

Conditions

• Hepatocellular Carcinoma

Interventions

Type	Name	Description
BIOLOGICAL	AlloStim® immunotherapy	3 cycles of intradermal and intravenous administrations
DRUG	FOLFOX regimen	Comparative arm: Physician Choice of FOLFOX4 chemotherapy
DRUG	Sorafenib	Comparative arm: Physician Choice of Sorafenib
DRUG	Lenvatinib	Comparative Arm: Physician's Choice of Levantinib

Timeline

• Start date: 2023-08-01
• Primary completion: 2026-11-01
• Completion: 2026-12-01
• First posted: 2021-09-02
• Last updated: 2025-10-23

Locations

8 sites across 2 countries: Malaysia, Thailand

Regulatory

• FDA-regulated drug study