NCT05595473

A Phase 1/2a, Open-label, Multicenter, Dose Escalation and Dose Expansion Study Evaluating the Safety, Tolerability and Efficacy of RZ-001 in Combination with Valganciclovir in Subjects with Hepatocellular Carcinoma

Active, enrolled Phase 1/Phase 2 Last updated 31 October 2024

What this trial tests

Phase 1/Phase 2 trial testing RZ-001 Dose 1 in Hepatocellular Carcinoma in 42 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

29 July 2022

Primary endpoint
1 March 2029

1 May 2029

Quick facts

Lead sponsor	Rznomics, Inc.
Phase	Phase 1/Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	42
Start date	29 July 2022
Primary completion	1 March 2029
Estimated completion	1 May 2029
Sites	5 locations across South Korea

Drugs / interventions tested

• RZ-001 Dose 1
• RZ-001 Dose 2
• RZ-001 Dose 3
• RZ-001 Dose 4

Conditions studied

• Hepatocellular Carcinoma — all drugs for Hepatocellular Carcinoma →

Sponsor

Rznomics, Inc. — full company profile →

Who can join

18 and older, any sex, with Hepatocellular Carcinoma. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of participants with Dose limiting toxicities (DLT) in Part 1 as graded by NCI-CTCAE
  Time frame: Day 1 to Day 28
• To determine safety and efficacy of RZ-001 by changes in overall response rate (ORR) for participants in part 2 as graded by RECIST v1.1 and mRECIST
  Time frame: Day 1 to Day 15
• To determine safety and efficacy of RZ-001 by changes in duration of response (DOR) in part 2 as graded by RECIST v1.1 and mRECIST
  Time frame: Day 1 to Day 15
• To determine safety and efficacy of RZ-001 by changes in Progression free survial (PFS) of participants in part 2 as graded by RECIST v1.1 and mRECIST
  Time frame: Day 1 to Day 15
• To determine safety and efficacy of RZ-001 by changes in overall survival (OS) of participants in part 2 as graded by RECIST v1.1 and mRECIST
  Time frame: Day 1 to Day 15
• Number of participants with adverse events (AEs) in part 1 and 2 as graded by NCI-CTCAE
  Time frame: Day 1 to Day 28

Sponsor's own description

This is first in human study to evaluate the safety, tolerability, immunogenicity, and preliminary clinical activity of RZ-001 when given to subjects with human telomerase reverse transcriptase (hTERT)-positive HCC.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Hepatocellular carcinoma: signaling pathways and therapeutic advances.
   Zheng J, Wang S, Xia L, Sun Z, et al · · 2025 · cited 142× · PMID 39915447 · DOI 10.1038/s41392-024-02075-w
2. Battle of the biopsies: Role of tissue and liquid biopsy in hepatocellular carcinoma.
   Lehrich BM, Zhang J, Monga SP, Dhanasekaran R. · · 2024 · cited 76× · PMID 38104635 · DOI 10.1016/j.jhep.2023.11.030
3. Molecular and immune landscape of hepatocellular carcinoma to guide therapeutic decision-making.
   Dhanasekaran R, Suzuki H, Lemaitre L, Kubota N, et al · · 2025 · cited 42× · PMID 37300379 · DOI 10.1097/hep.0000000000000513
4. Molecular and immune landscape of hepatocellular carcinoma for therapeutic development.
   Suzuki H, Mishra S, Paul S, Hoshida Y. · · 2025 · cited 8× · PMID 39639434 · DOI 10.17998/jlc.2024.12.02
5. Clinical research progress of telomerase targeted cancer immunotherapy: a literature review.
   Wang Y, Zhang X, Chen G, Shao M. · · 2024 · cited 6× · PMID 39145070 · DOI 10.21037/tcr-24-196
6. Gene Therapy Strategies for Hepatocellular Carcinoma (HCC): Current Landscape and Future Directions.
   Ermi AG, Younis RM, Rodriguez K, Sarkar D. · · 2025 · PMID 41300975 · DOI 10.3390/cancers17223608