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

Severe Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06378060
• Other study ID #: KM04
• Status: Recruiting
• Phase: Phase 2
• Start date: March 1, 2024
• Est. completion date: April 1, 2027

Study information

• Verified date: March 2024
• Source: Hematology department of the 920th hospital
• Contact: wang PI sanbin, Doctor
• Phone: 0871-64774206
• Email: Wangsanbin2022[@]126.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: April 1, 2027
• Est. primary completion date: April 1, 2026
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 3 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

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

Locations

Country	Name	City	State
China	920th Hospital of Joint Logistics Support Force of People's Liberation Army of China	Kunming	Yunnan

Sponsors (1)

Lead Sponsor	Collaborator
Hematology department of the 920th hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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]
Primary	Acute graft-versus-host disease incidence		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary	Transplantation-related motality		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary	Chronic graft-versus-host disease incidence		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary	incidence of Intensive fungal disease		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary	EB virus reactivation rate		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary	CMV reactivation rate		[Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]