Aplastic AnaemiaNCT07499284
A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia
A study testing Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells and Haploidentical hematopoietic cell transplantation in people with Aplastic Anaemia, run by The First Affiliated Hospital of Soochow University at 4 sites in China.
Trial summary
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and… Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Aplastic Anaemia
- What is being tested?
- Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells and Haploidentical hematopoietic cell transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 224 participants
- Where
- 4 sitesChina
- Study dates
- 1 Oct 2025to 30 Sep 2028
- Phase
- Interventional
- 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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Inclusion Criteria: (1)Aged ≥14 years. (2) Patients diagnosed with aplastic anemia meeting the diagnostic criteria of the Chinese Guidelines for the Diagnosis and Management of Aplastic Anemia (2024 Edition). (3) Patients who were eligible for allogeneic bone marrow or peripheral blood hematopoietic stem cell transplantation in accordance with the Clinical Application and Management Specifications for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition). (4) The patient understands the study protocol and voluntarily signs the informed consent form. (5) Life expectancy of ≥3 months; (6) Pre-transplant assessment meets the following criteria: Karnofsky Performance Status (KPS) score≥70, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score≤2, and Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score≤2.
Exclusion Criteria: (1)Patients with aplastic anemia secondary to malignancy treatment or those with concurrent active malignancy. (2) Women who are pregnant or lactating. (3) Patients with psychiatric or psychological disorders that preclude adequate compliance with the treatment protocol. (4) Patients with positive serologic testing for any of the four major infectious diseases (hepatitis B, hepatitis C, syphilis, HIV). (5) Patients with systemic infection or localized severe infection requiring active antimicrobial therapy. (6) Patients with significant dysfunction of major organs (e.g., heart, lung, liver, kidney). (7) Patients with a known allergy or hypersensitivity to any drug or component used in this study. (8) Patients who are currently participating in or plan to participate in any other clinical trial. (9) Any other condition deemed by the investigator to render the patient unsuitable for study participation.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China4 sites, 4 recruiting
- Recruiting
The First Affiliated Hospital of Zhejiang Chinese Medical University
Hangzhou, Zhejiang, China 310006
- Recruiting
Hematology Department, The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China 215006
- Recruiting
Suzhou Hongci Hematology Hospital
Suzhou, Jiangsu, China 215006
- Recruiting
The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, China 221006
About this study
Official title: A Multicenter, Randomized, Open-Label Study to Analyze the Efficacy of Unrelated Cord Blood Combined With Haploidentical Stem Cell Transplantation in Patients With Aplastic Anemia
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and immunity disorders.
Currently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte/lymphocyte globulin (ATG/ALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy.
Read more
Umbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation.
Currently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P < 0.0001).
Based on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV/EBV reactivation rate, donor chimerism dynamics, and immune reconstitution.
Read the detailed description
This study is designed to assess the safety and efficacy of haplo-cord HCT versus haplo HCT in patients with aplastic anemia, with particular emphasis on factors associated with treatment outcome and adverse events.
- Conditions
- Aplastic Anaemia
- Being tested
- Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells, Haploidentical hematopoietic cell transplantation
- Keywords
- Haplo-Cord HCT, Haplo HCT, Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cellsProcedure or surgery
- Donor Stem Cells Infusion (Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells) Day 0: Intravenous infuse a single unit of unrelated cord blood stem cells( TNC≤2×10⁷/kg, CD34+ cells≤0.8×10⁵/kg, HLA match≥5/10 loci, and either matched ABO type or a cord blood unit of type O). This is followed, 6 hours after the cord blood infusion, by the infusion of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Starting two days before transplantation (Day -2):Aciclovir (ACV): 250mg/㎡/8h.
Haploidentical hematopoietic cell transplantationProcedure or surgery
- Donor Stem Cell Infusion (Haploidentical hematopoietic cell transplantation) Day 0: Intravenous infuse of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Continuous medication starting from two days before transplantation (Day -2): Aciclovir (ACV): 250mg/㎡/8h.
| Study group | Role | What they receive |
|---|---|---|
| Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT | Experimental | Not described Receives: Procedure: Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells |
| Comparator group: 112 patients with aplastic anemia undergo haplo HCT | Active comparator | Not described Receives: Procedure: Haploidentical hematopoietic cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival rate
We estimated OS from the time of transplant until the date of death of any cause or last follow-up for patients still alive.
Measured: within 1 year following HSCT
Secondary outcomes (3)
The cumulative incidence of neutrophil engraftment and platelet engraftment
Neutrophil and platelet engraftment is defined as the first occurence of 3 consecutive days with an absolute neutrophil count of at least 0.5x10⁹/L and a platelet count of over 20x10⁹/L/L for 7 consecutive days without transfusion support.
Measured: on day 28+7 following HSCT
Disease Free Survival
We defined DFS as the time from the initiation of transplant to the occurrence of any treatment failure event (including graft failure or disease relapse requiring therapeutic intervention), or death from any cause, whichever occurred first. Patients who were alive and event-free at the last follow-up were censored.
Measured: within 1 year following HSCT
The cumulative incidence and grade of graft-versus-host disease (GVHD)
Graft-versus-host disease (GvHD) is a medical complication following the receipt of transplanted tissue from a genetically diferent person.
Measured: within 1 year following HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2026
- Study start1 Oct 2025
- Primary completion1 Sep 2028 (estimated)
- Study completion30 Sep 2028 (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.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 224 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Depei Wu, Study chair, The First Affiliated Hospital of Soochow UniversityXiaojin Wu, Study chair, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07499284
- Sponsor's protocol ID
- SOOCHOW-WXJ-2025-1075
- Status last verified
- Dec 2025
- First submitted
- 24 Mar 2026
- Last updated
- 30 Mar 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 30 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-Label Study to Analyze the Efficacy of Unrelated Cord Blood Combined With Haploidentical Stem Cell Transplantation in Patients With Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07499284
- Acronym / sponsor ID
- SOOCHOW-WXJ-2025-1075
- Conditions
- Aplastic Anaemia
- Keywords
- Haplo-Cord HCT, Haplo HCT, Aplastic Anemia
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 224 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Oct 2025 (actual)
- Primary completion
- 1 Sep 2028 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 30 Mar 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.
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and immunity disorders.
Currently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte/lymphocyte globulin (ATG/ALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy.
Umbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation.
Currently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P < 0.0001).
Based on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV/EBV reactivation rate, donor chimerism dynamics, and immune reconstitution.
Detailed description
This study is designed to assess the safety and efficacy of haplo-cord HCT versus haplo HCT in patients with aplastic anemia, with particular emphasis on factors associated with treatment outcome and adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT | Experimental | Procedure: Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells |
| Comparator group: 112 patients with aplastic anemia undergo haplo HCT | Active comparator | Procedure: Haploidentical hematopoietic cell transplantation |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells1. Donor Stem Cells Infusion (Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells) Day 0: Intravenous infuse a single unit of unrelated cord blood stem cells( TNC≤2×10⁷/kg, CD34+ cells≤0.8×10⁵/kg, HLA match≥5/10 loci, and either matched ABO type or a cord blood unit of type O). This is followed, 6 hours after the cord blood infusion, by the infusion of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Starting two days before transplantation (Day -2):Aciclovir (ACV): 250mg/㎡/8h. | Procedure or surgery | Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT |
| Haploidentical hematopoietic cell transplantation1. Donor Stem Cell Infusion (Haploidentical hematopoietic cell transplantation) Day 0: Intravenous infuse of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Continuous medication starting from two days before transplantation (Day -2): Aciclovir (ACV): 250mg/㎡/8h. | Procedure or surgery | Comparator group: 112 patients with aplastic anemia undergo haplo HCT |
Primary outcome
Overall survival rate
Time frame: within 1 year following HSCT
We estimated OS from the time of transplant until the date of death of any cause or last follow-up for patients still alive.
3 secondary outcomes
The cumulative incidence of neutrophil engraftment and platelet engraftment
Time frame: on day 28+7 following HSCT
Disease Free Survival
Time frame: within 1 year following HSCT
The cumulative incidence and grade of graft-versus-host disease (GVHD)
Time frame: within 1 year following HSCT
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 30 Mar 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.
Depei Wu study chair
The First Affiliated Hospital of Soochow University
Xiaojin Wu study chair
The First Affiliated Hospital of Soochow University
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells and Haploidentical hematopoietic cell transplantation in people with Aplastic Anaemia, run by The First Affiliated Hospital of Soochow University at 4 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: looking for participants now, as of the sponsor's last update on 30 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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.
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 Xiaojin Wu (+8613057493105, wuxiaojin@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival rate (time frame: within 1 year following HSCT). 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 30 Mar 2026.