A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 with Valganciclovir (VGCV) in Subjects with Hepatocellular Carcinoma

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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05595473

Enrollment

Registered

2022-10-27

Start date

2022-07-29

Completion date

2029-05-31

Last updated

2024-11-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hepatocellular Carcinoma

Brief summary

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.

Detailed description

The study will be conducted in 2 parts. Part 1 is a dose escalation study which aims to explore the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) after the single escalating dose of the intratumoral (IT) injection given through 4 different cohorts. Part 2 will consist of dose expansion, exploring clinical activity for the optimal fixed dose based on the results of Part 1.

Interventions

DRUGRZ-001 Dose 1

RZ-001 Dose 1 and VGCV

DRUGRZ-001 Dose 2

RZ-001 Dose 2 and VGCV

DRUGRZ-001 Dose 3

RZ-001 Dose 3 and VGCV

DRUGRZ-001 Dose 4

RZ-001 Dose 4 and VGCV

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Adult males and females * Hepatocellular carcinoma diagnosis (BCLC stage B or C) * hTERT positive expression confirmed during the screening period * ECOG score of 0 or 1 * Child-Pugh score of A to B7 * Life expectancy \>= 3 months

Exclusion criteria

* Moderate or severe ascites * History of hepatic encephalopathy * Carcinomas other than HCC * Current or history of HIV positive * Not suitable for inclusion judged by the investigator

Design outcomes

Primary

Measure	Time frame
Number of participants with adverse events (AEs) in part 1 and 2 as graded by NCI-CTCAE	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	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	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	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	Day 1 to Day 15
Number of participants with Dose limiting toxicities (DLT) in Part 1 as graded by NCI-CTCAE	Day 1 to Day 28

Secondary

Measure	Time frame
To determine safety and efficacy of RZ-001 by changes in duration of response (DOR) of the partipants in Part 1 as graded by RECIST v1.1 and mRECIST	Day 1 to Day 15
To determine safety and efficacy of RZ-001 by changes in Progression free survial (PFS) of the partipants in Part 1 as graded by RECIST v1.1 and mRECIST	Day 1 to Day 15
To determine safety and efficacy of RZ-001 by changes in overall survival (OS) of the partipants in Part 1 as graded by RECIST v1.1 and mRECIST	Day 1 to Day 15
To determine safety and efficacy of RZ-001 by changes in overall response rate (ORR) of the partipants in Part 1 as graded by RECIST v1.1 and mRECIST	Day 1 to Day 15

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026