Clinical trial NCT06378060: Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Severe Aplastic Anemia
Keywords: Severe Aplastic Anemia
Modified transplantation system
Ruxolitinib
Melphalan
Lead sponsor: Hematology department of the 920th hospital
Sponsor class: OTHER
First posted: 2024-04-22
Enrollment: 30
Sex: ALL
Minimum age (years): 3
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: overall survival (OS)
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
measure: Acute graft-versus-host disease incidence
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary outcomes:
measure: Transplantation-related motality
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
measure: Chronic graft-versus-host disease incidence
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
measure: incidence of Intensive fungal disease
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
measure: EB virus reactivation rate
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
measure: CMV reactivation rate
timeFrame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Other outcomes:
Locations:
city: Kunming
country: China
facility: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
state: Yunnan
Study officials:
References: