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

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

Study Overview

• Status: Recruiting
• Conditions: Severe Aplastic Anemia
• Intervention / Treatment: Drug: Modified transplantation system

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.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Wang PI sanbin, Doctor; Phone Number: 0871-64774206; Email: Wangsanbin2022@126.com

Study Locations

• China
  • Yunnan
    • Kunming, Yunnan, China, 650000
      • Recruiting
      • 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
      • Contact: Wang PI sanbin, Doctor; Phone Number: 0871-64774206; Email: Wangsanbin2022@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. 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;
2. Age 3-65 years old;
3. Weight 10Kg-100Kg;
4. Eastern Cooperative Oncology Group (ECOG) score ≤3;
5. 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);
6. No severe infection;
7. Subjects voluntarily participated in this clinical trial and signed the informed consent.

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental group	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
overall survival (OS)	2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Acute graft-versus-host disease incidence		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

Secondary Outcome Measures

Outcome Measure	Time Frame
Transplantation-related motality	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Chronic graft-versus-host disease incidence	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
incidence of Intensive fungal disease	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
EB virus reactivation rate	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
CMV reactivation rate	[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

Collaborators and Investigators

• Sponsor: Hematology department of the 920th hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2024
• Primary Completion (Estimated): April 1, 2026
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: April 17, 2024
• First Submitted That Met QC Criteria: April 19, 2024
• First Posted (Actual): April 22, 2024

Study Record Updates

• Last Update Posted (Actual): April 22, 2024
• Last Update Submitted That Met QC Criteria: April 19, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Ruxolitinib; Melphalan; Severe Aplastic Anemia; Modified transplantation system
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: KM04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No