NCT06378060

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Hematology department of the 920th hospital · Academic / Other
• Sex: All
• Age: 3 Years – 65 Years
• Healthy volunteers: Accepted

Summary

The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.

Detailed description

ObjectiveTo evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen in aplastic anemia. Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. We designed this study to improve the conditioning regimen and optimize the GHVD prevention measures to improve the transplant success rate and prolong patient survival, while minimizing the occurrence of GVHD and reducing the recurrence rate of the disease.30 patients with aplastic anemia were planned to be enrolled.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Modified transplantation system	Conditioning regimen: fludarabine 30mg/m2×5\~6d,cyclophosphamide 50mg/kg×2d, melphalan 100mg/m2×1d aGVHD prophylaxis: PTCY 25mg/kg×2d, ATG 4.5mg/kg total, MMF 15mg/kg +5d\~+35d, Ruxolitinib 5mg bid -1d\~+50d and 2.5mg bid +51d\~+110d, CSA

Timeline

• Start date: 2024-03-01
• Primary completion: 2026-04-01
• Completion: 2027-04-01
• First posted: 2024-04-22
• Last updated: 2024-04-22

Locations

1 site across 1 country: China