Actively Recruiting
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
Led by Hematology department of the 920th hospital · Updated on 2024-04-22
30
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a modified allogeneic hematopoietic stem cell transplantation (allo-HSCT) regimen for patients with severe aplastic anemia, a condition where the bone marrow fails to produce enough blood cells. This study aims to improve the success rate and patient survival by optimizing the treatment and reducing complications such as graft-versus-host disease (GVHD). It is a phase 2 clinical trial led by the Hematology department of the 920th hospital, enrolling 30 participants aged 3 to 65 years. The treatment involves a modified conditioning regimen including fludarabine, cyclophosphamide, and melphalan before transplantation. To prevent acute GVHD, the regimen includes PTCY, ATG, MMF, ruxolitinib, and cyclosporine (CSA) administered over a specified timeline before and after transplantation. This single-arm study evaluates this improved transplant system to enhance safety and efficacy in severe aplastic anemia patients. Participants will be monitored for two years after transplantation to assess overall survival and the incidence of acute and chronic GVHD. Additional evaluations include transplantation-related mortality, infections such as fungal diseases, and viral reactivations like EB virus and CMV. The study involves regular clinical assessments, laboratory tests, and safety monitoring to track outcomes and adverse events during this period.
CONDITIONS
Brief Title
Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients diagnosed with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, or aplastic anemia requiring allogeneic hematopoietic stem cell transplantation
- Age between 3 and 65 years old
- Weight between 10 kg and 100 kg
- Eastern Cooperative Oncology Group (ECOG) performance status score of 3 or less
- No major organ injury (heart ejection fraction over 45%; bilirubin less than twice the upper normal limit; AST and ALT less than three times the upper normal limit; serum creatinine less than twice the upper normal limit)
- No severe infection
- Voluntary participation with signed informed consent
You will not qualify if you...
- Patients with nonhematologic diseases who are not eligible or do not wish to receive transplantation
- Expected survival of less than 1 month
- Previous autologous or allogeneic hematopoietic stem cell transplantation
- Pregnant patients
- Severe mental or neurological disorders affecting consent or reporting adverse events
- Other conditions deemed inappropriate for enrollment by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 110 days
Participants receive a modified allogeneic hematopoietic stem cell transplantation regimen including conditioning drugs and graft-versus-host disease prophylaxis.
Multiple visits during treatment period for drug administration and monitoring
Duration - Up to 2 years after transplantation
Participants are monitored for safety, efficacy, and transplant outcomes including survival and graft-versus-host disease incidence for up to 2 years after transplantation.
Regular visits during follow-up period for assessments
Trial Site Locations
Total: 1 location
1
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, China, 650000
Actively Recruiting
Research Team
W
wang PI sanbin, Doctor
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here