NCT06039436

Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT

Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired Severe Aplastic Aplasia Receiving Single Cord Blood Transplant: A Multi-center, Single-arm Study

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 72 (estimated)
• Sponsor: Anhui Provincial Hospital · Other Government
• Sex: All
• Age: 1 Year – 50 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).

Detailed description

Hematopoietic stem cell transplantation (HSCT) from HLA-compatible sibling donors is the standard preferred therapy for Severe Aplastic Anemia (SAA). At present, HLA-compatible donors in China fail to meet the needs of patients for transplantation. Umbilical Cord blood transplantation (UCBT) is an alternative treatment for patients with emergency conditions who lack compatible sibling donors, have failed immunosuppressant therapies or cannot wait for unrelated fully compatible donors. However, implantation failure is one of the main problems in UCBT for SAA due to the low cord blood stem cells. Nowadays, there is no consensus on the optimal conditioning regimen for UCBT treatment of SAA, and whether ATG should be added to the conditioning regimen is still controversial. Early clinical studies in UCBT have shown that the sufficient ATG in the conditioning regimen will lead to a decrease rate of implantation rate and affect survival of UCBT ultimately. However, a range of recent studies have found that with the addition of low-dose ATG, T cell reconstitution seems to outperform bone marrow or peripheral blood stem cell transplantation. Therefore, how to properly use ATG to both retain its effective GVHD prevention effect and reduce its impact on immune reconstruction has important clinical significance for improving the transplantation efficacy. In this study, a multicenter clinical study will be conducted to observe the safety and efficacy of UCBT with low dose ATG in the treatment of SAA and the long-term quality of life of patients.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Anti-Thymocyte Globulin (Rabbit)	Indications for trial stopping: subjects occur pregnancy events, severe organ function impairments, or emergency events related or unrelated to treatment; or subjects are unwilling to continue participating in the clinical trial.

Timeline

• Start date: 2023-10-01
• Primary completion: 2025-04-30
• Completion: 2025-05-31
• First posted: 2023-09-15
• Last updated: 2023-10-24

Locations

1 site across 1 country: China