Clinical trial NCT05595473: A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 with Valganciclovir (VGCV) in Subjects with Hepatocellular Carcinoma
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Hepatocellular Carcinoma
Lead sponsor: Rznomics, Inc.
Sponsor class: OTHER
First posted: 2022-10-27
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Number of participants with Dose limiting toxicities (DLT) in Part 1 as graded by NCI-CTCAE
timeFrame: Day 1 to Day 28
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
measure: Number of participants with adverse events (AEs) in part 1 and 2 as graded by NCI-CTCAE
timeFrame: Day 1 to Day 28
Secondary outcomes:
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
measure: 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
timeFrame: Day 1 to Day 15
Other outcomes:
Locations:
city: Deagu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea, Seoul St. Mary's Hospital
Study officials:
References: