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
Overview
- Phase
- Phase 1
- Intervention
- RZ-001 Dose 1
- Conditions
- Hepatocellular Carcinoma
- Sponsor
- Rznomics, Inc.
- Enrollment
- 42
- Locations
- 5
- Primary Endpoint
- Number of participants with Dose limiting toxicities (DLT) in Part 1 as graded by NCI-CTCAE
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
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.
Investigators
Eligibility Criteria
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
Arms & Interventions
Cohort 1
Intervention: RZ-001 Dose 1
Cohort 2
Intervention: RZ-001 Dose 2
Cohort 3
Intervention: RZ-001 Dose 3
Cohort 4
Intervention: RZ-001 Dose 4
Outcomes
Primary Outcomes
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
Secondary Outcomes
- 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)