NCT06677255

A Phase I/II Trial of VUM02 Injection for Steroid-refractory Acute Graft-versus-host Disease (SR-aGvHD) Treatment

A Phase I/II Clinical Trial to Evaluate the Safety, Tolerability and Preliminary Efficacy of VUM02 Injection in the Treatment of Patients With Steroid-refractory Acute Graft-versus-host Disease (SR-aGvHD)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 149 (estimated)
• Sponsor: Wuhan Optics Valley Vcanbiopharma Co., Ltd. · Industry
• Sex: All
• Age: 14 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

It is a phase I/II clinical study to evaluate the safety, tolerability and preliminary efficacy of VUM02 Injection in patients with acute graft-versus-host disease (aGvHD) who have failed systemic steroid therapy. VUM02 Injection (human umbilical cord-derived mesenchymal stromal /stem cells, hUC-MSC) is an off-the-shelf allogeneic cell therapy product comprising culture-expanded mesenchymal stromal /stem cells derived from the human umbilical cord tissue. The product is cryopreserved with the cell concentration of 5 x 10\^6 cells/mL. Patients with grade II to IV aGvHD who have failed systemic steroid therapy (i.e. patients with steroid-refractory aGvHD (SR-aGvHD)), will be recruited into this study. This study consists of two phases, a dose-escalation phase (phase I) and a dose-expansion phase (phase II).

Detailed description

The Phase I study consists of a single-dose escalation Phase 1a and a multiple-dose escalation Phase 1b. * An open-label, single-dose escalation Phase 1a study is to evaluate the safety and tolerability of a single intravenous infusion of VUM02 Injection for SR-aGvHD treatment. The study follows an accelerated titration design with 3 dose levels (1×10\^6cells/kg, 2×10\^6cells/kg, 3×10\^6cells/kg). * An open-label, multiple-dose escalation Phase 1b study follows a traditional "3 + 3" design，with 3 dose levels of VUM02 administration twice weekly for 4 consecutive weeks, to evaluate the occurrence of dose-limiting toxicity (DLT) events from the first dose to 28 days after the last dose in subjects. After the multiple-dose escalation study to assess the tolerability and safety of VUM02 is completed, the dose expansion study will be initiated. Phase II is an open-label, randomized, parallel-controlled, multiple-dose expansion study. Two dose levels will be selected to evaluate the efficacy and safety of multiple dose administration by the investigator and the sponsor according to the results of the Phase 1b study. Subjects who meet the criteria are randomly assigned in a 1:1:1 ratio to receive the corresponding treatment in 3 groups, the study group 1 (VUM02-dose 1 + best available therapy (BAT) ), study group 2 (VUM02-dose 2 + BAT), and the control group (only BAT). Subjects in the study groups will receive the corresponding dose of VUM02 Injection, twice a week for 4 consecutive weeks for a total of 8 doses, on the basis of the best available therapy. All subjects are assessed for efficacy by day 28 after the first dose. In this study, ORR at day 28 after the first dose is used as the primary endpoint to investigate the efficacy of VUM02 Injection in the treatment of SR-aGvHD. Phase II study consists of four periods, a screening period (14 days), a VUM02 treatment period (4 weeks), a follow-up period (follow-up until death, consent withdrawal, or day 180±15 after the first dose, whichever occurs first), and a long-term follow-up period (after completion of the last visit of the follow-up period, entry into the long-term follow-up period).

Conditions

• Steroid-refractory Acute Graft-versus-host Disease

Interventions

Type	Name	Description
DRUG	VUM02 Injection (UC-MSCs)+BAT	The dose-escalation phase (phase I): Single dose escalation Phase 1a study: 3 dose levels of a single IV infusion; Multiple dose escalation Phase 1b study: 3 dose levels of twice weekly for 4 consecutive week. The dose-expansion phase (phase II): -Two dose groups will be selected by the investigator and the sponsor based on the results of the Phase 1b study, twice a week for 4 consecutive weeks for a total of 8 doses.
OTHER	The control group with Best Available Therapy (BAT)	According to BAT scheme, the drug administration is determined by PI according to the condition of the patients.

Timeline

• Start date: 2025-01-01
• Primary completion: 2026-12-01
• Completion: 2028-12-01
• First posted: 2024-11-06
• Last updated: 2024-11-06

Locations

1 site across 1 country: China