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

NCT06102525 | Recruiting Phase 1 FDA Drug

• 1 other identifier: RZ-001-201
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Glioblastoma

Outcome Measures

Primary Outcomes (5)

• Number of dose limiting toxicities (DLTs)

  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

  Day 1 to Day 28

• Number of participants with treatment-related adverse events as assessed by NCI-CTCAE

  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

  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)

  Day 1 to Day 15

Secondary Outcomes (7)

• Change in concentration of serum vascular endothelial growth factor (VEGF)

  Day 1 to Day 28

• Change in concentration of serum anti-adenovirus antibody

  Day 1 to Day 28

• Overall response rate (ORR)

  Day 1 to Day 15

• Duration of response (DOR)

  Day 1 to Day 15

• Progression-free survival (PFS) per modified Response Assessment for Neuro-Oncology (mRANO)

  Day 1 to Day 15

Other Outcomes (5)

• Concentration of adenovirus DNA in Plasma at specified timepoints

  Day 1 to Day 28

• Change in concentration of serum anti-adenovirus antibody

  Day 1 to Day 28

• Change in concentration of serum cytokines

  Day 1 to Day 28

Study Arms (6)

Part 1 Cohort 1

EXPERIMENTAL

RZ-001 Dose 1 and VGCV

Drug: RZ-001; Combination Product: VGCV

Part 1 Cohort 2

EXPERIMENTAL

RZ-001 Dose 2 and VGCV

Drug: RZ-001; Combination Product: VGCV

Part 1 Cohort 3

EXPERIMENTAL

RZ-001 Dose 3 and VGCV

Drug: RZ-001; Combination Product: VGCV

Part 1 Cohort 4

EXPERIMENTAL

RZ-001 Dose 4 and VGCV

Drug: RZ-001; Combination Product: VGCV

Part 1 Cohort 5

EXPERIMENTAL

RZ-001 Dose 5 and VGCV

Drug: RZ-001; Combination Product: VGCV

Part 2

EXPERIMENTAL

RZ-001 Dose 6 and VGCV

Drug: RZ-001; Combination Product: VGCV

Interventions

RZ-001 DRUG

Recombinant adenovirus harboring the modified ribozyme construct with HSV-tk as a therapeutic transgene

Also known as: Ad-ECRT-122T

Part 1 Cohort 1; Part 1 Cohort 2; Part 1 Cohort 3; Part 1 Cohort 4; Part 1 Cohort 5; Part 2

VGCV COMBINATION_PRODUCT

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.

Also known as: Valganciclovir

Part 1 Cohort 1; Part 1 Cohort 2; Part 1 Cohort 3; Part 1 Cohort 4; Part 1 Cohort 5; Part 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rznomics, Inc.: lead

Study Sites (6)

The Catholic University of Korea, Incheon St. Mary's Hospital

Incheon, 21431, South Korea

RECRUITING

Gachon University Gil Medical Center

Incheon, 21565, South Korea

RECRUITING

Seoul National University Bundang Hospital

Seongnam-si, 13620, South Korea

RECRUITING

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

RECRUITING

Asan Medical Center

Seoul, 05505, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

MeSH Terms

Conditions

Glioblastoma

Interventions

Valganciclovir

Condition Hierarchy (Ancestors)

Astrocytoma; Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Ganciclovir; Acyclovir; Guanine; Hypoxanthines; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Central Study Contacts

Rznomics Inc.

CONTACT

+82317068730; rznomics@rznomics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2023
• First Posted: October 26, 2023
• Study Start: October 8, 2024
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): May 1, 2029
• Last Updated: September 5, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

KR (6)