A Phase 2 interventional study of Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) in Aplastic Anaemia, sponsored by Anhui Provincial Hospital. Not yet recruiting at 1 site in China. Open to participants aged 1 Year to 50 Years. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Anhui Provincial Hospital · Phase 2, Interventional, and Prevention
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of Aplastic Anemia (AA)
Severe aplastic anemia (SAA) and transfusion-dependent non-severe aplastic anemia (TD-NSAA) are bone marrow failure disorders for which allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-matched sibling donor is the preferred curative treatment. For patients without a suitable matched sibling donor, single-unit umbilical cord blood transplantation (UCBT) is an alternative therapeutic option. However, graft failure remains a major challenge because of the limited number of hematopoietic stem cells in umbilical cord blood.
The optimal conditioning regimen for UCBT in patients with SAA/TD-NSAA has not been established. The use of antithymocyte globulin (ATG) in conditioning remains controversial. Previous studies suggested that conventional-dose ATG might reduce engraftment after UCBT, whereas more recent studies indicate that low-dose ATG may improve immune reconstitution while maintaining graft-versus-host disease (GVHD) prophylaxis.
This is a multicenter, single-arm clinical study designed to evaluate the safety and efficacy of a low-dose ATG-containing conditioning regimen followed by single-unit UCBT in patients with SAA or TD-NSAA. The study aims to further validate findings from a previous single-center study and to assess transplant outcomes, engraftment, immune reconstitution, GVHD, survival, and other clinical outcomes in a multicenter setting.
348 studies on the registry are indexed under Anemia, Aplastic; 100 are open to participants now.
This study's planned enrollment of 59 is above the median of 39 across 271 interventional studies indexed under Anemia, Aplastic.
Browse Anemia, Aplastic studies →Anhui Provincial Hospital is the lead sponsor of 131 studies on the registry; 97 are open to participants now.
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Drug: Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
Participants with aplastic anemia will receive a low-dose rabbit anti-thymocyte globulin (rATG)-containing conditioning regimen followed by single-unit umbilical cord blood transplantation (sUCBT). The conditioning regimen will consist of either rATG/fludarabine/cyclophosphamide/total body irradiation (rATG/Flu/Cy/TBI) or rATG/fludarabine/busulfan/cyclophosphamide (rATG/Flu/Bu/Cy), according to institutional practice and clinical conditions. Rabbit ATG (Thymoglobulin®) will be administered intravenously at a fixed dose of 1 mg/kg/day for 2 consecutive days from day -8 to day -7 before transplantation (total dose 2 mg/kg). The other conditioning agents will be administered according to the selected conditioning regimen.
The incidence of grade II to IV aGVHD
The primary endpoint of the study will be the development of grade II to IV aGVHD in the first 100 days post-transplant. Acute GVHD was graded according to the Mount Sinai Acute GVHD International Consortium criteria.
Time frame: Day 0 to Day 100 after transplantation
Incidence of Neutrophil Engraftment
The cumulative incidence of neutrophil engraftment. Neutrophil engraftment is defined as the first of three consecutive days with an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L.
Time frame: Day 0 to Day 42 after transplantation
Incidence of Platelet Engraftment
The cumulative incidence of platelet engraftment without platelet transfusion support.
Time frame: Day 0 to Day 60 after transplantation
Time to Platelet Engraftment
Time from transplantation to platelet engraftment, defined as the first of seven consecutive days with a platelet count ≥20 × 10⁹/L without platelet transfusion.
Time frame: From transplantation until platelet engraftment or Day 365 after transplantation, whichever occurs first
Cumulative Incidence of Platelet recovery
The cumulative incidence of platelet count ≥50 × 10⁹/L without platelet transfusion.
Time frame: Day 0 to Day 120 after transplantation
Incidence of Grade III-IV Acute GVHD
The proportion of participants who develop Grade III-IV acute graft-versus-host disease (aGVHD).
Time frame: Day 0 to Day 100 after transplantation
Incidence of Chronic GVHD
The proportion of participants who develop chronic graft-versus-host disease (cGVHD), diagnosed according to standard diagnostic criteria.
Time frame: Day 0 to Day 365 after transplantation
Immune Reconstitution
Immune reconstitution assessed by serum immunoglobulin levels (IgA, IgG, and IgM) and lymphocyte subset analysis, including total lymphocytes, T cells, CD4⁺ T cells, CD8⁺ T cells, regulatory T cells, B cells, NK cells, activated CD4⁺ T cells, activated CD8⁺ T cells, CD8⁺ effector memory T cells, CD8⁺ effector T cells, regulatory B cells, naïve B cells, and memory B cells.
Time frame: At 1, 2, 3, 6, and 12 months after transplantation
Transplant-Related Mortality (TRM)
The proportion of participants who die from causes other than disease progression or relapse following transplantation.
Time frame: Day 0 to Day 180 after transplantation
Incidence of Infection
The proportion of participants who develop documented bacterial, viral, or fungal infections after transplantation.
Time frame: Day 0 to Day 365 after transplantation
Disease-Free Survival (DFS)
Disease-free survival is defined as the time from transplantation to disease relapse, death from any cause, or the last follow-up, whichever occurs first.
Time frame: Day 0 to Day 365 after transplantation
Overall Survival (OS)
Overall survival is defined as the time from transplantation to death from any cause or the last follow-up.
Time frame: Day 0 to Day 365 after transplantation
Plan to share: No
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Studies of the drug tested here: Antilymphocyte Serum