RecruitingPhase 2NCT06378060

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


Sponsor

Hematology department of the 920th hospital

Enrollment

30 participants

Start Date

Mar 1, 2024

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 3 YearsMax Age: 65 Years

Inclusion Criteria7

  • Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;
  • Age 3-65 years old;
  • Weight 10Kg-100Kg;
  • Eastern Cooperative Oncology Group (ECOG) score ≤3;
  • No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
  • No severe infection;
  • Subjects voluntarily participated in this clinical trial and signed the informed consent.

Exclusion Criteria6

  • patients with nonhematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
  • patients with an expected survival of less than 1 month;
  • patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
  • pregnant patients;
  • patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
  • other conditions that the investigator determines to be inappropriate for enrollment.

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Interventions

DRUGModified 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


Locations(1)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, China

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