Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT

NCT07885982 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: CBAA001
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 1

Brief 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 Aplastic Anemia (AA)

Conditions

Aplastic Anaemia

Outcome Measures

Primary Outcomes (1)

• 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.

  Day 0 to Day 100 after transplantation

Secondary Outcomes (11)

• Incidence of Neutrophil Engraftment

  Day 0 to Day 42 after transplantation

• Incidence of Platelet Engraftment

  Day 0 to Day 60 after transplantation

• Time to Platelet Engraftment

  From transplantation until platelet engraftment or Day 365 after transplantation, whichever occurs first

• Cumulative Incidence of Platelet recovery

  Day 0 to Day 120 after transplantation

• Incidence of Grade III-IV Acute GVHD

  Day 0 to Day 100 after transplantation

Study Arms (1)

low-dose ATG-containing conditioning regimen

EXPERIMENTAL

Drug: Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)

Interventions

Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) DRUG

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.

low-dose ATG-containing conditioning regimen

Eligibility Criteria

• Age: 1 Year - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of severe aplastic anemia (SAA) according to the Camitta criteria, transfusion-dependent non-severe aplastic anemia (TD-NSAA), or inherited bone marrow failure syndrome (IBMFS) with an indication for allogeneic hematopoietic stem cell transplantation (HSCT).
• Age 1 to 50 years, regardless of sex.
• No available HLA-matched sibling donor or 10/10 HLA-matched unrelated donor.
• Eligible for single-unit unrelated umbilical cord blood transplantation (UCBT) using a cord blood unit that meets the protocol-defined HLA matching and minimum cell dose requirements.
• Negative donor-specific anti-HLA antibodies (DSA) against the selected cord blood unit before transplantation.
• Adequate cardiac, hepatic, renal, and pulmonary function.
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
• Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score 0-2.
• Serum ferritin \<1000 μg/L, or prior iron chelation therapy if serum ferritin exceeds this threshold.
• Female participants of childbearing potential must have a negative pregnancy test before enrollment and agree to use effective contraception during treatment and for 12 months after transplantation.
• Ability to understand the study requirements and provision of written informed consent by the participant or the participant's legally authorized representative.

You may not qualify if:

• Diagnosis of myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH), or other hematologic disorders causing pancytopenia.
• Previous allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation.
• Active, uncontrolled bacterial, viral, or fungal infection despite appropriate antimicrobial therapy.
• Human immunodeficiency virus (HIV) infection or active hepatitis B or hepatitis C infection.
• Pregnancy or breastfeeding.
• History of malignancy within 3 years before transplantation, except adequately treated basal cell carcinoma or carcinoma in situ of the cervix.
• Treatment with rabbit antithymocyte globulin (rATG) within 2 weeks before transplantation.
• Known hypersensitivity to rabbit antithymocyte globulin (rATG).
• Active substance abuse, uncontrolled psychiatric illness, cognitive impairment, or any condition that may interfere with study participation or protocol compliance.
• Participation in another interventional clinical trial within 3 months before enrollment.
• Any medical, psychological, or social condition that, in the opinion of the investigator, would make the participant unsuitable for the study or interfere with study participation or follow-up.

Sponsors & Collaborators

• Anhui Provincial Hospital: lead

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Hefei, Anhui, 230036, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Antilymphocyte Serum; thymoglobulin

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Central Study Contacts

Xiaoyu Zhu, Ph.D

CONTACT

15255456091; xiaoyuz@ustc.edu.cn

Bingbing Yan

CONTACT

15993691727; bing0415@mail.ustc.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 20, 2026
• First Posted: October 21, 2026
• Study Start (Estimated): August 1, 2027
• Primary Completion (Estimated): April 30, 2029
• Study Completion (Estimated): April 30, 2029
• Last Updated: October 21, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)