A Prospective, Open Label, Dose-escalation, SAD/MAD, Multicenter, 24weeks, Phase I/IIa Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of Investigational Product (VM-001) in Patients With Graft-versus-host Disease(GvHD)

The present study is multi center, phase I/IIa clinical trial to evaluate the safety and pharmacodynamics of investigational Product (VM-001) in patients with graft-versus-host disease(GvHD). A total of 12 subjects (Part 1 SAD[3 cohort(2 subjects/cohort)], Part 2 MAD[2 cohort(3subjects/cohort)]) are recruited.

Study Overview

• Status: Not yet recruiting
• Conditions: Graft Versus Host Disease
• Intervention / Treatment: Biological: VM-001 1X10^6 cells/kg; Biological: VM-001 3X10^6 cells/kg; Biological: VM-001 5X10^6 cells/kg; Biological: VM-001 1X10^6 cells/kg two dose; Biological: VM-001 1X10^6 cells/kg four dose

• Study Type: Interventional
• Enrollment (Anticipated): 12
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Hyo Jung Park, Ph.D.; Phone Number: 82-70-4348-7457; Email: parkhyojung@vigencell.com
• Study Contact Backup: Name: Hyun-Jung Sohn, Ph.D.; Phone Number: 82-70-4348-7527; Email: sohnhj@vigencell.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Men and women ≥19 years of age
• Acute GvHD or chronic GvHD after hematopoietic stem cell transplant
• Subjects who no longer have available standard treatment.
• ANC≥1,000cells./mm^3
• AST, ALT, total bilirubin less than 3 times the upper limit of normal
• serum creatinine less than 1.5 times the upper limit of normal

Exclusion Criteria:

• Received an anti-thymocyte globulin(ATG) within 14 days before enrollment
• FCV or FEV less than 70%
• Any uncontrolled infection or active infection requiring ongoing systemic treatment
• Received an investigational agent within 6 months before enrollment.
• Evidence of bleeding diathesis or coagulopathy.
• Active hepatitis C virus (HCV) or hepatitis B virus (HBV)
• Breastfeeding or pregnant.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part 1 Single Ascending Dose, Cohort 1; Cohort 1 : 1X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the two subjects: Begin enrollment for Cohort 2.	Biological: VM-001 1X10^6 cells/kg; Administration: Inject intravenously single dose
Experimental: Part 1 Single Ascending Dose, Cohort 2; Cohort 2 : 3X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the two subjects: Begin enrollment for Cohort 3.	Biological: VM-001 3X10^6 cells/kg; Administration: Inject intravenously single dose
Experimental: Part 1 Single Ascending Dose, Cohort 3; Cohort 3 : 5X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP.	Biological: VM-001 5X10^6 cells/kg; Administration: Inject intravenously single dose
Experimental: Part 2 Multiple Ascending Dose, Cohort 1; Cohort 1 : two doses in total, 1X10^6 cells/kg per dose, weekly The safety of 3 subjects is evaluated for 4 week after two dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the three subjects: Begin enrollment for Cohort 2.	Biological: VM-001 1X10^6 cells/kg two dose; Administration: Inject intravenously 1X10^6 cells/kg per dose, two dose in total, weekly
Experimental: Part 2 Multiple Ascending Dose, Cohort 2; Cohort 2 : four doses in total, 1X10^6 cells/kg per dose, weekly The safety of 3 subjects is evaluated for 8 week after two dose of IP.	Biological: VM-001 1X10^6 cells/kg four dose; Administration: Inject intravenously 1X10^6 cells/kg per dose, four dose in total, weekly

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Tolerated Dose (MTD) of VM-001 [ Time Frame: 1 week ]	First 7-day treatment dose limiting toxicities (DLT) graded according to CTCAE in the MTD-determining population in Phase 1 based on the number of participants with adverse effects as measure of tolerability at various dose levels	7 day cycle

Collaborators and Investigators

• Sponsor: ViGenCell Inc.
• Investigators: Principal Investigator: Chang-Ki Min, MD, Ph.D., The Catholic University of Korea

Study record dates

Study Major Dates

• Study Start (Anticipated): November 20, 2022
• Primary Completion (Anticipated): November 20, 2024
• Study Completion (Anticipated): November 20, 2025

Study Registration Dates

• First Submitted: March 2, 2022
• First Submitted That Met QC Criteria: March 2, 2022
• First Posted (Actual): March 11, 2022

Study Record Updates

• Last Update Posted (Actual): March 11, 2022
• Last Update Submitted That Met QC Criteria: March 2, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: VM-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: No
• Studies a U.S. FDA-regulated device product: No