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

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.

Study Overview

• Status: Recruiting
• Conditions: Hepatocellular Carcinoma
• Intervention / Treatment: Drug: RZ-001 Dose 1; Drug: RZ-001 Dose 2; Drug: RZ-001 Dose 3; Drug: RZ-001 Dose 4

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 Type: Interventional
• Enrollment (Anticipated): 42
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Rznomics Inc.; Phone Number: +82317068730; Email: rznomics@rznomics.com
• Study Contact Backup: Name: Hyunjin Yoon; Phone Number: +82-31-701-8735; Email: hjyoon@rznomics.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 06351
    • Recruiting
    • Samsung Medical Center
    • Contact: Yong Han Paik

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1	Drug: RZ-001 Dose 1; RZ-001 Dose 1 and VGCV
Experimental: Cohort 2	Drug: RZ-001 Dose 2; RZ-001 Dose 2 and VGCV
Experimental: Cohort 3	Drug: RZ-001 Dose 3; RZ-001 Dose 3 and VGCV
Experimental: Cohort 4	Drug: RZ-001 Dose 4; RZ-001 Dose 4 and VGCV

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Rznomics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 29, 2022
• Primary Completion (Anticipated): March 1, 2029
• Study Completion (Anticipated): May 1, 2029

Study Registration Dates

• First Submitted: September 7, 2022
• First Submitted That Met QC Criteria: October 25, 2022
• First Posted (Actual): October 27, 2022

Study Record Updates

• Last Update Posted (Actual): October 27, 2022
• Last Update Submitted That Met QC Criteria: October 25, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Adenocarcinoma; Neoplasms, Glandular and Epithelial; Digestive System Neoplasms; Liver Diseases; Liver Neoplasms; Carcinoma; Carcinoma, Hepatocellular
• Other Study ID Numbers: RZ-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No