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)

ViGenCell

Status and phase: Not yet enrolling; Phase 2; Phase 1

Conditions: Graft Versus Host Disease

Treatments: Biological: VM-001 3X10^6 cells/kg; Biological: VM-001 1X10^6 cells/kg four dose; Biological: VM-001 1X10^6 cells/kg; Biological: VM-001 1X10^6 cells/kg two dose; Biological: VM-001 5X10^6 cells/kg

Study type: Interventional

Funder types: Industry

Identifiers

NCT05276076

VM-201

Details and patient eligibility

About

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.

Enrollment: 12 estimated patients

Sex: All

Ages: 19+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

12 participants in 5 patient groups

Part 1 Single Ascending Dose, Cohort 1

Experimental group

Description:

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.

Treatment:

Biological: VM-001 1X10^6 cells/kg

Part 1 Single Ascending Dose, Cohort 2

Experimental group

Description:

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.

Treatment:

Biological: VM-001 3X10^6 cells/kg

Part 1 Single Ascending Dose, Cohort 3

Experimental group

Description:

Cohort 3 : 5X10\^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP.

Treatment:

Biological: VM-001 5X10^6 cells/kg

Part 2 Multiple Ascending Dose, Cohort 1

Experimental group

Description:

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.

Treatment:

Biological: VM-001 1X10^6 cells/kg two dose

Part 2 Multiple Ascending Dose, Cohort 2

Experimental group

Description:

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.

Treatment:

Biological: VM-001 1X10^6 cells/kg four dose