Modified stem cell transplantation for severe aplastic anemia

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

Phase 2 Interventional Hematology department of the 920th hospital · NCT06378060

This study is testing a new way of doing stem cell transplants for people with severe aplastic anemia to see if it can help them live longer and reduce complications.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment30 (estimated)
Ages3 Years to 65 Years
SexAll
SponsorHematology department of the 920th hospital Academic / other
Locations1 site (Kunming, Yunnan)
Trial IDNCT06378060 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen specifically for patients with severe aplastic anemia. The study aims to enhance the success rate of the transplantation process by improving the conditioning regimen and optimizing measures to prevent graft-versus-host disease (GVHD). A total of 30 patients aged 3 to 65 years with various hematological diseases will be enrolled to assess the outcomes of this modified approach. The goal is to prolong patient survival while minimizing complications associated with the procedure.

Who should consider this trial

Good fit: Ideal candidates for this study are patients aged 3-65 years with severe aplastic anemia or other hematological diseases requiring allogeneic stem cell transplantation.

Not a fit: Patients with non-hematologic diseases or those with an expected survival of less than one month may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could significantly improve survival rates and quality of life for patients with severe aplastic anemia.

How similar studies have performed: Other studies have shown promising results with modified transplantation regimens, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Kunming, Yunnan

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Severe Aplastic AnemiaModified transplantation systemRuxolitinibMelphalan
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.