Natural Killer (NK) Cell Therapy in Locally Advanced HCC

Vaxcell Bio

Status and phase: Completed; Phase 2

Conditions: Locally Advanced Hepatocellular Carcinoma

Treatments: Biological: Vax-NK/HCC

Study type: Interventional

Funder types: Industry

Identifiers

NCT05040438

Vax-NK/HCC-201

Details and patient eligibility

About

This Phase 2a trial will evaluate the safety and efficacy of NK cell therapy combined with the hepatic artery infusion chemotherapy (HAIC) in patients with intermediate and/or locally advanced hepatocellular carcinoma (HCC). We hypothesized that 5-fluorouracil (FU) with immunomodulatory functions would relieve the immunosuppressive microenvironment from the myeloid-derived suppressor cells (MDSCs), thereby enhancing the anti-tumor activity of NK cells. Thus, the subsequent infusion of autologous NK cells (VAX-NK/HCC) following HAIC treatment may further improve the anti-tumor activity in patients with advanced HCC.

Full description

Primary Objective I. To assess the objective response rate (ORR) of administering VAX-NK/HCC, autologous NK cells combined with HAIC in patients with locally advanced HCC.

Secondary Objectives I. To assess the efficacy of administering VAX-NK/HCC combined with HAIC. II. To assess the safety of administering VAX-NK/HCC combined with HAIC. III. To assess the immune responses of administering VAX-NK/HCC combined with HAIC.

OUTLINE: This is a Phase 2a study. Patients receive HAIC treatment every 4 week for up to 4 cycles followed by ex-vivo expanded autologous NK cell infusions. The NK cell treatment repeats every 4 weeks for up to 2 cycles in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients will be followed until the disease progression.

Enrollment: 17 patients

Sex: All

Ages: 19+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects with intermediate and/or locally advanced HCC histologically confirmed by biopsy or by typical radiological findings.
• Subjects who were not suitable for or failed curative treatments such as surgical resection, local ablation therapy, transarterial chemoembolization (TACE), sorafenib, atezolizumab, bevacizumab, etc.
• Child-Pugh liver function class A or B.
• Subjects' ECOG performance status of 0 or 1.
• The presence of macrovascular invasion.
• Adequate liver, renal, and hematologic functions.

Exclusion criteria

• Subjects who received the immune cell-based therapy within 6 months before the screening visit.
• Subjects with a history of a malignancy other than HCC within the last 5 years, liver transplantation, and hypersensitivity to 5-FU or cisplatin.
• Subjects with extra-hepatic metastases.
• Subjects who have ongoing autoimmune disease.
• Female subjects who are pregnant or lactating or women of child-bearing potential but unable to take adequate contraception.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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17 participants in 1 patient group

Autologous NK cell infusion combined with HAIC

Experimental group

Description:

HAIC of 5-FU (500 mg/m2, Q4W) and cisplatin (15 mg/m2, Q4W) will be administered for up to 4 cycles to patients with locally advanced HCC. Subjects who achieved sustained SD or better based on the mRECIST criteria after 2nd cycle of HAIC will be enrolled to receive 1x10\^9 cells VAX-NK/HCC infusion.

Treatment:

Biological: Vax-NK/HCC