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

August 29, 2025 updated by: Rznomics, Inc.

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 Glioblastoma

This is a Phase 1/2a, open-label study to evaluate the safety, tolerability, immunogenicity, and preliminary clinical activity of RZ-001 administered in combination with VGCV in subjects with hTERT-positive GBM.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The study consists of 2 parts: a dose-escalation part (Part 1) and a dose-expansion part (Part 2).

Part 1 consists of dose escalation exploring MTD/RP2D for intratumoral (IT) injection.

Part 2 will consist of dose expansion exploring clinical activity for the optimal fixed dose based on the results of Part 1.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
43

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • South Korea
      • Incheon, South Korea, 21565
        • Recruiting
        • Gachon University Gil Medical Center
        • Contact:
          • Gachon University Gil Medical Center
      • Incheon, South Korea, 21431
        • Recruiting
        • The Catholic University of Korea, Incheon St. Mary's Hospital
        • Contact:
          • The Catholic University of Korea, Incheon St. Mary's Hospital
      • Seongnam-si, South Korea, 13620
        • Recruiting
        • Seoul National University Bundang Hospital
        • Contact:
          • Seoul National University Bundang Hospital
      • Seoul, South Korea, 05505
        • Recruiting
        • Asan Medical Center
        • Contact:
          • Asan Medical Center
      • Seoul, South Korea, 03722
        • Recruiting
        • Severance Hospital, Yonsei University Health System
        • Contact:
          • Severance Hospital, Yonsei University Health System
      • Seoul, South Korea, 06351
        • Recruiting
        • Samsung Medical Center
        • Contact:
          • Samsung Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Adult males and females
  • Histologically-confirmed grade 4 astrocytoma, GBM, per The 2021 WHO Classification of CNS Tumors.
  • hTERT positive expression confirmed during the screening period
  • ECOG score of ≤ 2
  • KPS ≥ 60
  • Life expectancy ≥ 3 months

Exclusion Criteria:

  • Diagnosis of other malignant tumors within 5 years prior to RZ-001 administration.
  • Have extracranial metastases of the tumor cells
  • Current or history of HIV positive
  • Not suitable for inclusion judged by the investigator

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Part 1 Cohort 1
RZ-001 Dose 1 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir
Experimental: Part 1 Cohort 2
RZ-001 Dose 2 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir
Experimental: Part 1 Cohort 3
RZ-001 Dose 3 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir
Experimental: Part 1 Cohort 4
RZ-001 Dose 4 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir
Experimental: Part 1 Cohort 5
RZ-001 Dose 5 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir
Experimental: Part 2
RZ-001 Dose 6 and VGCV
Drug: RZ-001
Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene
Other Names:
  • Ad-ECRT-122T
Combination product: VGCV
VGCV, used in a subject after RZ-001 administration, is a nucleoside analog that is metabolized by HSV-tk and other cellular kinases to form the cytotoxic nucleotide analog ganciclovir triphosphate. An approved oral VGCV will be used in the proposed clinical study of RZ-001.
Other Names:
  • Valganciclovir

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of dose limiting toxicities (DLTs)
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Maximum tolerated dose (MTD) or maximum administered dose (MAD) dose(MAD) and select the recommended Phase 2 dose (RP2D) of RZ-001 in combination with VGCV
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Number of participants with treatment-related adverse events as assessed by NCI-CTCAE
Time Frame: Day 1 to Day 28
Adverse events (AEs) as characterized by type, number, severity (as graded by National Cancer Institute Common Terminology Criteria for Adverse Events [NCI-CTCAE]), timing, seriousness, and relationship to RZ-001
Day 1 to Day 28
Number of participants with significant laboratory abnormalities as assessed by NCI-CTCAE
Time Frame: Day 1 to Day 28
Clinically significant laboratory abnormalities as characterized by type, frequency, severity (as graded by NCI-CTCAE), timing, and relationship to RZ-001
Day 1 to Day 28
Overall survival (OS)
Time Frame: Day 1 to Day 15
Day 1 to Day 15

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Overall survival (OS)
Time Frame: Day 1 to Day 15
Day 1 to Day 15
Change in concentration of serum vascular endothelial growth factor (VEGF)
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Change in concentration of serum anti-adenovirus antibody
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Overall response rate (ORR)
Time Frame: Day 1 to Day 15
Day 1 to Day 15
Duration of response (DOR)
Time Frame: Day 1 to Day 15
Day 1 to Day 15
Progression-free survival (PFS) per modified Response Assessment for Neuro-Oncology (mRANO)
Time Frame: Day 1 to Day 15
Day 1 to Day 15
Neurologic function assessment using the Neurologic Assessment in Neuro-Oncology (NANO) scale ranging from 0 to 3 in each assessment domain
Time Frame: Day 1 to Day 15
Day 1 to Day 15

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in concentration of serum anti-adenovirus antibody
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Concentration of adenovirus DNA in Plasma at specified timepoints
Time Frame: Day 1 to Day 28
Day 1 to Day 28
Change in concentration of serum cytokines
Time Frame: Day 1 to Day 28
Serum cytokines including interleukins 1 (IL-1), IL-6, IL-10, IL-27, interferon gamma (IFN-γ), tumor necrosis factor alpha (TNF-α)
Day 1 to Day 28
Concentration of biomarker in peripheral blood
Time Frame: Day 1 to Day 28
Activation of immune cell subsets (including but not limited to cluster of differentiation 3 [CD3], CD4, CD8, B cell, natural killer [NK] cell)
Day 1 to Day 28
Concentration of biomarker in fresh tumor biopsy tissue
Time Frame: Day 1 to Day 28
Tumor-related RNA and T cell infiltration and activation
Day 1 to Day 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Rznomics, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Doo Sik Kong, Samsung Medical Center
  • Principal Investigator: Chang Ki Hong, Asan Medical Center
  • Principal Investigator: Chae-yong Kim, Seoul National University Bundang Hospital
  • Principal Investigator: Gi-Taek Yee, Gachon University Gil Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 8, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 23, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 22, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 26, 2023

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 5, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 29, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RZ-001-201

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

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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