Locally Advanced Hepatocellular CarcinomaPhase 2NCT05040438
Natural Killer (NK) Cell Therapy in Locally Advanced HCC
A Phase 2 study testing Vax-NK/HCC in people with Locally Advanced Hepatocellular Carcinoma, run by Vaxcell Bio, Co., Ltd. at 1 site in South Korea.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Locally Advanced Hepatocellular Carcinoma
- What is being tested?
- Vax-NK/HCC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteSouth Korea
- Study dates
- 15 Oct 2019to 14 Sep 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2a Study Using Natural Killer (NK) Cell Therapy Combined With Hepatic Artery Infusion Chemotherapy (HAIC) in Patients With Locally Advanced Hepatocellular Carcinoma
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.
Read the detailed 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.
- Being tested
- Vax-NK/HCC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vax-NK/HCCBiological
autologous NK cells expanded ex vivo.
| Study group | Role | What they receive |
|---|---|---|
| Autologous NK cell infusion combined with HAIC | Experimental | 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. Receives: Biological: Vax-NK/HCC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR) of administering VAX-NK/HCC combined with HAIC
ORR will be measured as the proportion of patients with a best overall response of complete response (CR) and partial response (PR) of administering VAX-NK/HCC combined with HAIC.
Measured: average 6 months
Secondary outcomes (9)
Disease control rate (DCR) of administering VAX-NK/HCC combined with HAIC
ORR will be measured as the proportion of patients with a best overall response of complete response (CR), partial response (PR), and stable disease (SD) of administering VAX-NK/HCC combined with HAIC.
Measured: average 6 months
Time to progression (TTP) of administering VAX-NK/HCC combined with HAIC
TTP will be measured by time to progression, defined as time from enrollment to disease progression.
Measured: average 6 months
Overall survival (OS) of administering VAX-NK/HCC combined with HAIC
OS will be measured as time from enrollment to death due to any cause.
Measured: average 12 months
Quality of Life of administering VAX-NK/HCC combined with HAIC
The assessment will be performed using the Korean versions of European Organization for Research and Treatment of Cancer (EORTC) Questionnaire 30 (QLQ-C30) consisting of 30 items. Total score: Range 0-100.
Measured: average 6 months
Adverse Events (AEs) and Serious Adverse Events (SAEs) of administering VAX-NK/HCC combined with HAIC
The assessment will be measured by determining the number of patients that experience AEs and SAEs graded according to the NCI-CTCAE (Version 4.0)
Measured: average 6 months
The proportions of T and NK cells
This will be measured by determining the relative percentages of CD4+CD8+ T cells and CD3- CD56+ NK cells in patients' peripheral blood.
Measured: average 6 months
The lymphocyte/monocyte ratio (LMR)
LMR will be calculated by dividing the absolute lymphocyte count by the absolute monocyte count in patients' peripheral blood.
Measured: average 6 months
The NK cell cytotoxicity
This will be measured by determining percent cell lysis of target cells (K562) in patients' peripheral blood.
Measured: average 6 months
The serum cytokine levels
The serum concentrations of IFN-γ, IL-10, and TGF-β will be measured in patients' serum using the Enzyme-Linked immunosorbent assays.
Measured: average 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2021
- Study start15 Oct 2019
- Primary completion14 Sep 2023
- Study completion14 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Locally Advanced Hepatocellular CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vaxcell Bio, Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Seon-Ah Ha, Ph.D., Study director, VaxCell Biotherapeutics Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05040438
- Sponsor's protocol ID
- Vax-NK/HCC-201
- Status last verified
- May 2023
- First submitted
- 26 Aug 2021
- Last updated
- 30 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Study Using Natural Killer (NK) Cell Therapy Combined With Hepatic Artery Infusion Chemotherapy (HAIC) in Patients With Locally Advanced Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT05040438
- Acronym / sponsor ID
- Vax-NK/HCC-201
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Vaxcell Bio, Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Oct 2019 (actual)
- Primary completion
- 14 Sep 2023 (actual)
- Study completion
- 14 Sep 2023 (actual)
- First posted
- 10 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous NK cell infusion combined with HAIC | Experimental | Biological: Vax-NK/HCCHAIC 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. |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vax-NK/HCCautologous NK cells expanded ex vivo. | Biological | Autologous NK cell infusion combined with HAIC |
Primary outcome
Objective Response Rate (ORR) of administering VAX-NK/HCC combined with HAIC
Time frame: average 6 months
ORR will be measured as the proportion of patients with a best overall response of complete response (CR) and partial response (PR) of administering VAX-NK/HCC combined with HAIC.
9 secondary outcomes
Disease control rate (DCR) of administering VAX-NK/HCC combined with HAIC
Time frame: average 6 months
Time to progression (TTP) of administering VAX-NK/HCC combined with HAIC
Time frame: average 6 months
Overall survival (OS) of administering VAX-NK/HCC combined with HAIC
Time frame: average 12 months
Quality of Life of administering VAX-NK/HCC combined with HAIC
Time frame: average 6 months
Adverse Events (AEs) and Serious Adverse Events (SAEs) of administering VAX-NK/HCC combined with HAIC
Time frame: average 6 months
The proportions of T and NK cells
Time frame: average 6 months
The lymphocyte/monocyte ratio (LMR)
Time frame: average 6 months
The NK cell cytotoxicity
Time frame: average 6 months
The serum cytokine levels
Time frame: average 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Seon-Ah Ha, Ph.D. study director
VaxCell Biotherapeutics Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vax-NK/HCC in people with Locally Advanced Hepatocellular Carcinoma, run by Vaxcell Bio, Co., Ltd. at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) of administering VAX-NK/HCC combined with HAIC (time frame: average 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2024.