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

NCT ID: NCT06378060

Last Updated: 2024-04-22

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-03-01
• Study Completion Date: 2027-04-01

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Experimental group

Group Type: EXPERIMENTAL

Modified transplantation system

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Hematology department of the 920th hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Kunming, Yunnan, China

Site Status: RECRUITING

Countries

China

Central Contacts

wang PI sanbin, Doctor

Role: CONTACT

Wangsanbin2022@126.com

0871-64774206

Facility Contacts

Wang PI sanbin, Doctor

Role: primary

Wangsanbin2022@126.com

0871-64774206

Other Identifiers

KM04

Identifier Type: -

Identifier Source: org_study_id