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)
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Arms and Interventions
| 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
|
Outcome Measures
Primary Outcome Measures
- Maximum Tolerated Dose (MTD) of VM-001 [ Time Frame: 1 week ] [7 day cycle]
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
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- ViGenCell Inc.
Investigators
- Principal Investigator: Chang-Ki Min, MD, Ph.D., The Catholic University of Korea
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- VM-201