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

Phase 1

Active, not recruiting

• Conditions: Hepatocellular Carcinoma
• Interventions: Drug: RZ-001 Dose 1; Drug: RZ-001 Dose 2; Drug: RZ-001 Dose 3; Drug: RZ-001 Dose 4

• Registration Number: NCT05595473
• Lead Sponsor: Rznomics, Inc.

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.

Study Timeline

• Study Start: 2022-07-29
• Study First Submitted: 2022-09-07
• Study First Submitted QC: 2022-10-25
• Study First Posted: 2022-10-27
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-31
• Last Update Posted: 2024-11-04
• Primary Completion: 2029-03
• Study Completion: 2029-05

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 42

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	RZ-001 Dose 1	-
Cohort 2	RZ-001 Dose 2	-
Cohort 3	RZ-001 Dose 3	-
Cohort 4	RZ-001 Dose 4	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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
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

Trial Locations

Locations (5)

Kyungpook National University Hospital; 🇰🇷 Deagu, Korea, Republic of

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Severance Hospital; 🇰🇷 Seoul, Korea, Republic of

The Catholic University of Korea, Seoul St. Mary's Hospital; 🇰🇷 Seoul, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of