Application of mRNA Immunotherapy Technology in Hepatitis B Virus-related Refractory Hepatocellular Carcinoma

NCT ID: NCT05738447

Last Updated: 2023-02-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE1

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-15

Study Completion Date

2025-01-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to evaluate the efficacy and safety of mRNA vaccine for HBV-positive Advanced Hepatocellular Carcinoma.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Hepatocellular carcinoma is a common malignant tumor of the digestive system worldwide and is particularly prevalent in China. More than 80% of hepatocellular carcinoma patients in China have concomitant hepatitis B virus infection. However, there are limited effective treatments for hepatocellular carcinoma that have failed standard treatment, and the prognosis for these patients is poor. About 350 million people worldwide are chronically infected with the hepatitis B virus (HBV), and chronic HBV infection accounts for at least 50% of hepatocellular carcinoma cases worldwide. Therefore, anti-HBV can be a potential target for hepatocellular carcinoma.

The mRNA vaccines are a highly promising novel anti-tumor approach. The applicant team of this project has carried out research on raw materials and preparation process, vaccine stability, quality standard, delivery vector construction, and anti-tumor mechanism of action of mRNA immune formulation in the early stage. Now we have completed the optimized design of mRNA raw materials, and the construction and optimization of the mRNA delivery vector. The tumor therapeutic mRNA immune formulation with high efficiency, safety, scalable preparation, and quality control has been selected and preclinical evaluation has been completed to verify its safety and efficacy. The project has constructed mRNA vaccines, and no similar therapeutic method or product has been reported in the international arena.

This project proposes to conduct a phase I clinical study based on the previous study to include patients with advanced hepatocellular carcinoma who have failed second-line standard treatment or cannot receive standard treatment. A dose-escalation trial will be conducted, and one effective dose will be selected for a fixed-dose trial to explore the safety, tolerability, and efficacy of mRNA immunotherapy technology for clinical application. Project implementation is expected to benefit a wide range of hepatocellular carcinoma patients and improve their prognosis.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Liver Cancer Hepatocellular Carcinoma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Treatment Cohort

With 20ug as the starting point, the dose was increased using a dose escalation scheme. Each subject only received one corresponding dose, and the intramuscular injection was administered again every 7 days, and after 4 doses, the 5th dose was given after 1-month interval.

Group Type EXPERIMENTAL

HBV mRNA vaccine

Intervention Type BIOLOGICAL

With 20ug as the starting point, the dose was increased using a dose escalation scheme. Each subject only received one corresponding dose, and the intramuscular injection was administered again every 7 days, and after 4 doses, the 5th dose was given after 1-month interval.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

HBV mRNA vaccine

With 20ug as the starting point, the dose was increased using a dose escalation scheme. Each subject only received one corresponding dose, and the intramuscular injection was administered again every 7 days, and after 4 doses, the 5th dose was given after 1-month interval.

Intervention Type BIOLOGICAL

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Male or female patients: ≥ 18 years old; ≤ 70 years old;
2. Patients with HBV-positive advanced hepatocellular carcinoma after failure of second-line standard therapy (including PD-1 inhibitor therapy, chemotherapy, and anti-vascular targeted drugs);
3. HBsAg positive, regardless of whether the peripheral blood is positive for HBV DNA.
4. ECOG physical fitness score: 0\~1 points;
5. Estimated survival ≥ 3 months;
6. The main organs have good function, that is, the relevant examination indicators within random 14 days meet the following requirements:

1. Blood routine examination: hemoglobin ≥ 80 g/L (no blood transfusion within 14 days); Neutrophil count\> 1.5×109/L; Platelet count≥ 80×109/L;
2. Biochemical examination: total bilirubin ≤ 1.5× ULN (upper limit of normal); alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 2.5×ULN; If liver metastases are present, ALT or AST ≤ 5×ULN; Endogenous creatinine clearance

≥ 60 ml/min (Cockcroft-Gault formula);
3. Cardiac Doppler ultrasound assessment: left ventricular ejection fraction (LVEF) ≥50%.
7. Sign the informed consent form;
8. Good compliance, family members agree to cooperate with survival follow-up.

Exclusion Criteria

1. Participated in clinical trials of other drugs within 4 weeks;
2. The patient has a history of other tumors, unless it is cervical cancer in situ, treated cutaneous squamous cell carcinoma or bladder epithelial tumor or other malignant tumors that has received radical treatment (at least 5 years before enrollment)
3. Patients with uncontrolled cardiac clinical symptoms or diseases, such as heart failure above NYHA grade 2, unstable angina, myocardial infarction within 1 year, and clinically significant supraventricular or ventricular arrhythmias requiring treatment or intervention.
4. For female subjects: pregnant or lactating women.
5. The patient has active tuberculosis, bacterial or fungal infection (≥ grade 2 of NCI-CTC, 3rd edition); There is HIV infection with active HBV infection, HCV infection.
6. Those who have a history of psychotropic drug abuse and have mental disorders that cannot be remitted;
7. The subject has any active autoimmune disease or has a history of autoimmune disease (such as, but not limited to uveitis, enteritis, pituitary inflammation, nephritis, hyperthyroidism, hypothyroidism; Participants with vitiligo or who had complete remission of asthma in childhood and did not require any intervention in adulthood could be included; Participants in asthma requiring medical intervention with bronchodilators omitted).
8. According to the judgment of the investigator, there are concomitant diseases that seriously endanger the safety of patients or affect the completion of the patient's research.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

West China Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Xingchen Peng

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Xingchen Peng

Role: PRINCIPAL_INVESTIGATOR

West China Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

West China Hospital, Sichuan University

Chengdu, Sichuan, China

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

China

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Xingchen Peng

Role: CONTACT

+86 18980606753

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Xingchen Peng

Role: primary

+86 18980606753

References

Explore related publications, articles, or registry entries linked to this study.

Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I, Jemal A, Bray F. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. CA Cancer J Clin. 2021 May;71(3):209-249. doi: 10.3322/caac.21660. Epub 2021 Feb 4.

Reference Type BACKGROUND
PMID: 33538338 (View on PubMed)

Zhang BH, Yang BH, Tang ZY. Randomized controlled trial of screening for hepatocellular carcinoma. J Cancer Res Clin Oncol. 2004 Jul;130(7):417-22. doi: 10.1007/s00432-004-0552-0.

Reference Type BACKGROUND
PMID: 15042359 (View on PubMed)

Ganem D, Prince AM. Hepatitis B virus infection--natural history and clinical consequences. N Engl J Med. 2004 Mar 11;350(11):1118-29. doi: 10.1056/NEJMra031087. No abstract available.

Reference Type BACKGROUND
PMID: 15014185 (View on PubMed)

Parkin DM. The global health burden of infection-associated cancers in the year 2002. Int J Cancer. 2006 Jun 15;118(12):3030-44. doi: 10.1002/ijc.21731.

Reference Type BACKGROUND
PMID: 16404738 (View on PubMed)

Mittal S, El-Serag HB. Epidemiology of hepatocellular carcinoma: consider the population. J Clin Gastroenterol. 2013 Jul;47 Suppl(0):S2-6. doi: 10.1097/MCG.0b013e3182872f29.

Reference Type BACKGROUND
PMID: 23632345 (View on PubMed)

Chang MH, Chen CJ, Lai MS, Hsu HM, Wu TC, Kong MS, Liang DC, Shau WY, Chen DS. Universal hepatitis B vaccination in Taiwan and the incidence of hepatocellular carcinoma in children. Taiwan Childhood Hepatoma Study Group. N Engl J Med. 1997 Jun 26;336(26):1855-9. doi: 10.1056/NEJM199706263362602.

Reference Type BACKGROUND
PMID: 9197213 (View on PubMed)

Greenberg DP. Pediatric experience with recombinant hepatitis B vaccines and relevant safety and immunogenicity studies. Pediatr Infect Dis J. 1993 May;12(5):438-45. doi: 10.1097/00006454-199305000-00037.

Reference Type BACKGROUND
PMID: 8327313 (View on PubMed)

Liaw YF, Sung JJ, Chow WC, Farrell G, Lee CZ, Yuen H, Tanwandee T, Tao QM, Shue K, Keene ON, Dixon JS, Gray DF, Sabbat J; Cirrhosis Asian Lamivudine Multicentre Study Group. Lamivudine for patients with chronic hepatitis B and advanced liver disease. N Engl J Med. 2004 Oct 7;351(15):1521-31. doi: 10.1056/NEJMoa033364.

Reference Type BACKGROUND
PMID: 15470215 (View on PubMed)

Papatheodoridis GV, Lampertico P, Manolakopoulos S, Lok A. Incidence of hepatocellular carcinoma in chronic hepatitis B patients receiving nucleos(t)ide therapy: a systematic review. J Hepatol. 2010 Aug;53(2):348-56. doi: 10.1016/j.jhep.2010.02.035. Epub 2010 Apr 27.

Reference Type BACKGROUND
PMID: 20483498 (View on PubMed)

Papatheodoridis GV, Chan HL, Hansen BE, Janssen HL, Lampertico P. Risk of hepatocellular carcinoma in chronic hepatitis B: assessment and modification with current antiviral therapy. J Hepatol. 2015 Apr;62(4):956-67. doi: 10.1016/j.jhep.2015.01.002. Epub 2015 Jan 13.

Reference Type BACKGROUND
PMID: 25595883 (View on PubMed)

Isogawa M, Chung J, Murata Y, Kakimi K, Chisari FV. CD40 activation rescues antiviral CD8(+) T cells from PD-1-mediated exhaustion. PLoS Pathog. 2013;9(7):e1003490. doi: 10.1371/journal.ppat.1003490. Epub 2013 Jul 11.

Reference Type BACKGROUND
PMID: 23853599 (View on PubMed)

Virgin HW, Wherry EJ, Ahmed R. Redefining chronic viral infection. Cell. 2009 Jul 10;138(1):30-50. doi: 10.1016/j.cell.2009.06.036.

Reference Type BACKGROUND
PMID: 19596234 (View on PubMed)

Shouval D, Roggendorf H, Roggendorf M. Enhanced immune response to hepatitis B vaccination through immunization with a Pre-S1/Pre-S2/S vaccine. Med Microbiol Immunol. 2015 Feb;204(1):57-68. doi: 10.1007/s00430-014-0374-x. Epub 2015 Jan 4.

Reference Type BACKGROUND
PMID: 25557605 (View on PubMed)

Nishikawa J, Yoshiyama H, Iizasa H, Kanehiro Y, Nakamura M, Nishimura J, Saito M, Okamoto T, Sakai K, Suehiro Y, Yamasaki T, Oga A, Yanai H, Sakaida I. Epstein-barr virus in gastric carcinoma. Cancers (Basel). 2014 Nov 7;6(4):2259-74. doi: 10.3390/cancers6042259.

Reference Type BACKGROUND
PMID: 25386788 (View on PubMed)

Reinhard K, Rengstl B, Oehm P, Michel K, Billmeier A, Hayduk N, Klein O, Kuna K, Ouchan Y, Woll S, Christ E, Weber D, Suchan M, Bukur T, Birtel M, Jahndel V, Mroz K, Hobohm K, Kranz L, Diken M, Kuhlcke K, Tureci O, Sahin U. An RNA vaccine drives expansion and efficacy of claudin-CAR-T cells against solid tumors. Science. 2020 Jan 24;367(6476):446-453. doi: 10.1126/science.aay5967. Epub 2020 Jan 2.

Reference Type BACKGROUND
PMID: 31896660 (View on PubMed)

Wang F, Qin Z, Lu H, He S, Luo J, Jin C, Song X. Clinical translation of gene medicine. J Gene Med. 2019 Jul;21(7):e3108. doi: 10.1002/jgm.3108. Epub 2019 Jul 15.

Reference Type BACKGROUND
PMID: 31246328 (View on PubMed)

Fenton OS, Kauffman KJ, McClellan RL, Kaczmarek JC, Zeng MD, Andresen JL, Rhym LH, Heartlein MW, DeRosa F, Anderson DG. Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs. Angew Chem Int Ed Engl. 2018 Oct 8;57(41):13582-13586. doi: 10.1002/anie.201809056. Epub 2018 Sep 14.

Reference Type BACKGROUND
PMID: 30112821 (View on PubMed)

Miao L, Li L, Huang Y, Delcassian D, Chahal J, Han J, Shi Y, Sadtler K, Gao W, Lin J, Doloff JC, Langer R, Anderson DG. Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation. Nat Biotechnol. 2019 Oct;37(10):1174-1185. doi: 10.1038/s41587-019-0247-3. Epub 2019 Sep 30.

Reference Type BACKGROUND
PMID: 31570898 (View on PubMed)

Hou X, Zhang X, Zhao W, Zeng C, Deng B, McComb DW, Du S, Zhang C, Li W, Dong Y. Author Correction: Vitamin lipid nanoparticles enable adoptive macrophage transfer for the treatment of multidrug-resistant bacterial sepsis. Nat Nanotechnol. 2020 Jul;15(7):615. doi: 10.1038/s41565-020-0675-8.

Reference Type BACKGROUND
PMID: 32346117 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2022-1371

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.