Optimizing conditioning regimens for stem cell transplants in severe aplastic anemia
To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
This study is testing different treatment plans before stem cell transplants to see which one helps people with severe aplastic anemia do better after the procedure.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 160 (estimated) |
| Ages | 1 Year to 50 Years |
| Sex | All |
| Sponsor | Peking University People's Hospital Academic / other |
| Drugs / interventions | Cyclophosphamide, Fludarabine |
| Locations | 1 site (Beijing, Beijing) |
| Trial ID | NCT06069180 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the efficacy and safety of different conditioning regimens for hematopoietic stem cell transplantation (HSCT) in patients with severe aplastic anemia (SAA). It will compare the traditional regimens, Flu/Cy/ATG and Cy/ATG, with a modified regimen that includes busulfan to reduce the incidence of mixed chimerism and improve failure-free survival. The study is a prospective, multicenter, randomized controlled trial involving patients who have an HLA-matched donor. The goal is to establish a standardized conditioning regimen that enhances patient outcomes post-transplantation.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with severe aplastic anemia who have an HLA-matched sibling or unrelated donor and meet specific health criteria.
Not a fit: Patients over 50 years old or those with severe organ dysfunction or active infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved survival rates and better outcomes for patients undergoing HSCT for severe aplastic anemia.
How similar studies have performed: Previous studies have suggested that modifying conditioning regimens can improve outcomes, indicating potential success for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Diagnosed as SAA/vSAA 2. Indication for hematopoietic stem cell transplantation 3. Available HLA matched sibling or unrelated donor 4. No active infection 5. No serious organ damage: liver and kidney function (ALT and AST \< 2.5 times normal value, normal renal function, no cardiac insufficiency) 6. Signed informed consent 7. High risk factors of mixed chimerism, at least one of the following 1. Age \< 18 years old 2. Ferritin level ≥2500ng/ml before transplantation Exclusion Criteria: 1. Age \> 50 years old 2. ECOG≥3 3. Active infections that were difficult to control 4. Severe liver and kidney dysfunction 5. Mental illness 6. Not signing the informed consent 7. pregnant or lactating women 8. Any condition considered by the investigators to be unsuitable for enrollment
Where this trial is running
Beijing, Beijing
- Deparment of Hematology, Peking University People's Hospital — Beijing, Beijing, China (Recruiting)
Study contacts
- Principal investigator: Xiao-Jun Huang — Peking University People's Hospital
- Study coordinator: Zheng-Li Xu, M.D.
- Email: xuzhengli0202@163.com
- Phone: +8613501338951
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.