NCT06164288

Safety and Efficacy Study of hAESCs Therapy for aGVHD

A Phase I Study to Evaluate the Safety and Efficacy of Human Amniotic Epithelial Stem Cell（hAESCs）Injection Treatment for the Patients With Refractory aGVHD After Allogeneic Hematopoietic Cell Transplantation (HCT)

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Allogeneic hematopoietic stem cell transplantation (allo-HSCT), a process in which hematopoietic stem cells from a donor are injected into the recipient's body, are the treatment of choice for many hematologic malignancies. Graft-versus-host disease (GVHD) is a common and important complication after allogeneic HSCT. GVHD is a major obstacle to the success of HSCT treatment and a leading cause of death after HSCT treatment. Hormone therapy is currently the standard treatment for aGVHD, i.e., the first-line treatment. However, 40%\~50% of aGVHD cannot be controlled by hormone therapy, and additional therapeutic intervention is required. According to the National Comprehensive Cancer Network (NCCN) clinical practice guidelines for hematopoietic stem cell transplantation - pre-transplant recipient evaluation and management of GVHD (2021.V3), the recommended drugs for second-line treatment of grade II\~IV aGVHD include: alemtuzumab, α-1 antitrypsin, antithymocyte globulin, basiliximab, calcineurin inhibitors, etanercept, extracorporeal photopheresis replacement therapy, infliximab, mammalian rapamycin target protein inhibitors, mycophenolate mofetil, Pentostatin, ruxolitinib, tocilizumab. Second-line treatment is based on retrospective data and there is no standard salvage therapy, which is reflected in the inconsistent treatment strategy for aGVHD across transplant centers. One of the biological functions of hAESCs in amniotic membranes in vivo is to exert reproductive immunomodulatory effects and protect the fetus from rejection by the maternal immune system, so hAESCs have natural immunomodulatory functions. hAESCs have significant inhibitory effects on T cells, antigen-presenting cells (APCs), natural killer (NK) cells, macrophages, neutrophils, B cells and other immune cells associated with organ damage during the pathogenesis of aGVHD, and hAESCs have great potential in the treatment of aGVHD. Therefore, the sponsor developed hAESCs injections intended for the treatment of aGVHD. The experimental drug in this study is hAESCs injection, which is intended to be used for the treatment of adult patients with grade III.\~IV. refractory aGVHD after hematopoietic stem cell transplantation, and to explore the safety and preliminary efficacy of its treatment.

Conditions

• GVHD,Acute

Interventions

Type	Name	Description
DRUG	Human Amniotic Epithelial Stem Cell Injection	The dose escalation is carried out according to the "3+3" ascending principle, and a total of 3 dose levels are set: 1. Dose Level 1: 1×106 cells/kg; 2. Dose Level 2: 2×106 cells/kg; 3. Dose Level 3: 3×106 cells/kg; Note: In this study, if the maximum tolerated dose (MTD) is not explored at dose level 3 (3×106 cells/kg), it will be discussed by the Safety Review Committee (SRC) to decide whether to proceed with further dose escalation or expansion to 6 patients.

Timeline

• Start date: 2024-01-01
• Primary completion: 2024-12-31
• Completion: 2025-06-30
• First posted: 2023-12-11
• Last updated: 2023-12-11