Aplastic AnaemiaPhase 2NCT07885982
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT
A Phase 2 study testing Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) in people with Aplastic Anaemia, run by Anhui Provincial Hospital at 1 site in China.
Trial 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) Read more about this study
- Who is it for?
- Ages 1 year to 50 years
- Conditions the study is for
- Aplastic Anaemia
- What is being tested?
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 59 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2027to 30 Apr 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, 230036
Xiaoyu Zhu, Ph.D, 15255456091, xiaoyuz@ustc.cn
About this study
Official title: Conditioning Regimen Containing Low-Dose Antithymocyte Globulin (ATG) for the Treatment of Aplastic Anemia Receiving Single-Unit Umbilical Cord Blood Transplantation: A Multicenter, Single-Arm Study
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)
Read the detailed description
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.
- Conditions
- Aplastic Anaemia
- Being tested
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| low-dose ATG-containing conditioning regimen | Experimental | Not described Receives: Drug: Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 0 to Day 100 after transplantation
Secondary outcomes (11)
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.
Measured: Day 0 to Day 42 after transplantation
Incidence of Platelet Engraftment
The cumulative incidence of platelet engraftment without platelet transfusion support.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Day 0 to Day 180 after transplantation
Incidence of Infection
The proportion of participants who develop documented bacterial, viral, or fungal infections after transplantation.
Measured: 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.
Measured: 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.
Measured: Day 0 to Day 365 after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start1 Aug 2027 (estimated)
- Primary completion30 Apr 2029 (estimated)
- Study completion30 Apr 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnaemiaStudies of the same condition, open and under way.
- More trials run by Anhui Provincial HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 59 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Anhui Provincial Hospital
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07885982
- Sponsor's protocol ID
- CBAA001
- Status last verified
- Jul 2026
- First submitted
- 20 Aug 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Conditioning Regimen Containing Low-Dose Antithymocyte Globulin (ATG) for the Treatment of Aplastic Anemia Receiving Single-Unit Umbilical Cord Blood Transplantation: A Multicenter, Single-Arm Study
- ClinicalTrials.gov ID
- NCT07885982
- Acronym / sponsor ID
- CBAA001
- Conditions
- Aplastic Anaemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 59 participants (estimated)
- Lead sponsor
- Anhui Provincial HospitalOther government
- Study start
- 1 Aug 2027 (estimated)
- Primary completion
- 30 Apr 2029 (estimated)
- Study completion
- 30 Apr 2029 (estimated)
- First posted
- 21 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| low-dose ATG-containing conditioning regimen | Experimental | Drug: Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) |
Who may qualify
- Ages
- 1 year to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | low-dose ATG-containing conditioning regimen |
Primary outcome
The incidence of grade II to IV aGVHD
Time frame: Day 0 to Day 100 after transplantation
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.
11 secondary outcomes
Incidence of Neutrophil Engraftment
Time frame: Day 0 to Day 42 after transplantation
Incidence of Platelet Engraftment
Time frame: Day 0 to Day 60 after transplantation
Time to Platelet Engraftment
Time frame: From transplantation until platelet engraftment or Day 365 after transplantation, whichever occurs first
Cumulative Incidence of Platelet recovery
Time frame: Day 0 to Day 120 after transplantation
Incidence of Grade III-IV Acute GVHD
Time frame: Day 0 to Day 100 after transplantation
Incidence of Chronic GVHD
Time frame: Day 0 to Day 365 after transplantation
Immune Reconstitution
Time frame: At 1, 2, 3, 6, and 12 months after transplantation
Transplant-Related Mortality (TRM)
Time frame: Day 0 to Day 180 after transplantation
Incidence of Infection
Time frame: Day 0 to Day 365 after transplantation
Disease-Free Survival (DFS)
Time frame: Day 0 to Day 365 after transplantation
Overall Survival (OS)
Time frame: Day 0 to Day 365 after transplantation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) in people with Aplastic Anaemia, run by Anhui Provincial Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaoyu Zhu, Ph.D (15255456091, xiaoyuz@ustc.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of grade II to IV aGVHD (time frame: Day 0 to Day 100 after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.