Aplastic AnemiasNCT06650553
Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study
A study testing Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group. in people with Aplastic Anemias, run by Shanxi Bethune Hospital at 1 site in China.
Trial summary
Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. Read more about this study
- Who is it for?
- Ages 6 to 75 years
- Conditions the study is for
- Aplastic Anemias
- What is being tested?
- Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 110 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2024to Jul 2027
- 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 6 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A patient age of 6-75 years
- Patients diagnosed with Severe Aplastic Anemia (SAA) , Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) or Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT
- Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study
Who cannot join
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
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, 1 recruiting
- Recruiting
Shanxi Bethune Hospital
Taiyuan, Shanxi, 030000
Tao Wang, Dr., 13835175119, wangtao99699@163.com
About this study
Official title: Clinical Study on Umbilical Cord Blood-Supported Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Aplastic Anemia
Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.
Read the detailed description
Aplastic anemia (aplastic anaemia，AA) is a syndrome of bone marrow hematopoietic dysfunction （bone marrow hematopoietic failure，BMF）with an annual incidence rate of approximately 0.74/100,000 people, affecting all age groups with a higher incidence rate in the elderly, and similar incidence rates between males and females. AA is divided into congenital and acquired types, with acquired AA being more common. The pathogenesis of primary acquired AA is mainly related to the abnormal activation of T lymphocytes, and genetic background may also play a role in the development and progression of AA. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the best treatment for AA, with a cure rate close to 90%. Sibling allogeneic hematopoietic stem cell transplantation (HLA-matched HSCT) is the preferred treatment plan, but it is not easy to find a fully matched sibling donor. For patients who relapse after immunosuppressive therapy (IST), haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is a good alternative, despite the risks of engraftment failure, infection, and graft-versus-host disease (GVHD). With the advancement of transplantation technology, the efficacy of haplo-HSCT is not significantly different from that of unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) and HLA-matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT). Studies have shown that haplo-HSCT has a higher survival rate in treating patients with severe aplastic anemia (SAA), and there is no significant difference compared to MSD-HSCT.To address the risk of complications associated with haplo-HSCT, recent research has indicated that co-transplantation with third-party umbilical cord blood cells can shorten the time for hematopoietic reconstitution and improve prognosis. The co-transplantation of third-party umbilical cord blood with bone marrow and peripheral blood hematopoietic stem cells has become a new transplantation option. Studies at home and abroad have shown that combined third-party transplantation can shorten the time for hematopoietic reconstitution and improve long-term survival rates. This study plans to adopt a prospective, open-label, parallel-controlled trial design to conduct a clinical study of non-related umbilical cord blood-assisted haploidentical allogeneic hematopoietic stem cell transplantation for the treatment of AA. The study will compare the umbilical cord blood combined with haploidentical transplantation plan with sibling fully matched transplantation, to assess the feasibility and safety of the umbilical cord blood combined with haploidentical transplantation plan, and to monitor the incidence of patient infections, acute/chronic GVHD incidence, transplant-related mortality (TRM), and event-free survival (EFS), in the hope of providing new practical experience in the field of AA treatment, and bringing dual benefits to patients and the social medical enterprise.
- Conditions
- Aplastic Anemias
- Being tested
- Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.
- Keywords
- Aplastic Anemias, Stem Cell Transplantation, Umbilical Cord Blood
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.Procedure or surgery
Conditioning regimen consisted of the following1)Severe aplastic anemia(SAA):FC-ATG:Flu 30 mg/m^2/d ×4d (- 7d- -4d), CTX 22.5 -25mg/kg/d × 4d (-7d- -4d), ATG 7.5mg in total（- 5d- -2d).2)Transfusion-dependent non-severe aplastic anemia（TD-NSAA) and Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA):BU/FC-ATG:Bu 0.8mg/kg(q6h -8d）+FC-ATG. The GVHD prophylaxis program was Application of Anti-CD25 Humanized Monoclonal Antibody 50mg (+1d,+4d);cyclosporine A + mycophenolate mofetil (MMF)+short course methotrexate (MTX).The infusion time of umbilical cord blood (UCB) stem cells in the UCB-assisted haploidentical hematopoietic stem cell transplantation (UCB-Haplo-HSCT) group: 4 hours before the infusion of peripheral blood stem cells.In the follow-up phase, outcome evaluation indicators must be collected according to the follow-up plan, ensuring data quality.
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study | Experimental | In patients with AA undergoing hematopoietic stem cell transplantation, the feasibility of the umbilical cord blood combined with haploidentical transplantation scheme is assessed by comparing it with sibling fully matched transplantation using a non-inferiority analysis method (with the time of neutrophil engraftment after transplantation as the primary efficacy indicator). At the same time, indicators such as the time of platelet engraftment, infection incidence (including CMV, EBV incidence), acute/chronic GvHD incidence, transplant-related mortality (TRM), event-free survival (EFS), and overall survival (OS) are monitored to evaluate the safety and effectiveness of this scheme. Receives: Procedure: Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to neutrophil engraftment
Neutrophil engraftment was defined as the first of three consecutive days on which the patient's absolute neutrophil count was 500/uL.
Measured: 60 days
Secondary outcomes (6)
Time to platelet engraftment
It was defined as independence from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10^9/L.
Measured: 60 days
Incidence of infections (including CMV and EBV incidence)
CMV DNAemia was defined as positive CMV-DNA in the blood when the copies exceeded 500 copies/ml.EBV DNAemia was defined as positive EBV-DNA in the blood when the copies exceeded 500 copies/ml.
Measured: 100 days,6 months,12 months,24 months
Overall survival rate (OS)
Overall survival is a secondary endpoint that will be measured as time from the start of treatment until death from any cause, or the last date the patient was known to be alive.
Measured: 24 months
Event-free survival (EFS)
Considering as event aGVHD, SOS, relapse and death
Measured: 24 months
Incidence of acute/chronic GvHD
aGVHD (acute GVHD) was defined according to the 1994 Consensus Conference on Acute GVHD Grading and graded from I to IV. Chronic GVHD (cGVHD) was graded as limited or extensive.
Measured: 24 months
Transplant-related mortality (TRM)
Transplant-related mortality (TRM) is defined as the proportion of patients who die as a direct or indirect result of the complications arising from the hematopoietic stem cell transplantation (HSCT) process, rather than from the underlying disease for which the transplant was performed. This includes deaths due to graft failure, severe infections, organ toxicity, graft-versus-host disease (GVHD), and other treatment-related complications.
Measured: 100 days,6 months,12 months,24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2024
- Study start1 Jun 2024
- Primary completionJun 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiasStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 110 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanxi Bethune Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tao Wang, Dr., Study director, Shanxi Bethune Hospital Regulatory Authority
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06650553
- Sponsor's protocol ID
- IIT-2024-018-FS02
- Status last verified
- Sep 2024
- First submitted
- 28 Sep 2024
- Last updated
- 14 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on Umbilical Cord Blood-Supported Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06650553
- Acronym / sponsor ID
- IIT-2024-018-FS02
- Conditions
- Aplastic Anemias
- Keywords
- Aplastic Anemias, Stem Cell Transplantation, Umbilical Cord Blood
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 110 participants (estimated)
- Lead sponsor
- Shanxi Bethune HospitalUniversity, hospital or other
- Study start
- 1 Jun 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 21 Oct 2024 (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 rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.
Detailed description
Aplastic anemia (aplastic anaemia，AA) is a syndrome of bone marrow hematopoietic dysfunction （bone marrow hematopoietic failure，BMF）with an annual incidence rate of approximately 0.74/100,000 people, affecting all age groups with a higher incidence rate in the elderly, and similar incidence rates between males and females. AA is divided into congenital and acquired types, with acquired AA being more common. The pathogenesis of primary acquired AA is mainly related to the abnormal activation of T lymphocytes, and genetic background may also play a role in the development and progression of AA. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the best treatment for AA, with a cure rate close to 90%. Sibling allogeneic hematopoietic stem cell transplantation (HLA-matched HSCT) is the preferred treatment plan, but it is not easy to find a fully matched sibling donor. For patients who relapse after immunosuppressive therapy (IST), haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is a good alternative, despite the risks of engraftment failure, infection, and graft-versus-host disease (GVHD). With the advancement of transplantation technology, the efficacy of haplo-HSCT is not significantly different from that of unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) and HLA-matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT). Studies have shown that haplo-HSCT has a higher survival rate in treating patients with severe aplastic anemia (SAA), and there is no significant difference compared to MSD-HSCT.To address the risk of complications associated with haplo-HSCT, recent research has indicated that co-transplantation with third-party umbilical cord blood cells can shorten the time for hematopoietic reconstitution and improve prognosis. The co-transplantation of third-party umbilical cord blood with bone marrow and peripheral blood hematopoietic stem cells has become a new transplantation option. Studies at home and abroad have shown that combined third-party transplantation can shorten the time for hematopoietic reconstitution and improve long-term survival rates. This study plans to adopt a prospective, open-label, parallel-controlled trial design to conduct a clinical study of non-related umbilical cord blood-assisted haploidentical allogeneic hematopoietic stem cell transplantation for the treatment of AA. The study will compare the umbilical cord blood combined with haploidentical transplantation plan with sibling fully matched transplantation, to assess the feasibility and safety of the umbilical cord blood combined with haploidentical transplantation plan, and to monitor the incidence of patient infections, acute/chronic GVHD incidence, transplant-related mortality (TRM), and event-free survival (EFS), in the hope of providing new practical experience in the field of AA treatment, and bringing dual benefits to patients and the social medical enterprise.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study | Experimental | Procedure: Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.In patients with AA undergoing hematopoietic stem cell transplantation, the feasibility of the umbilical cord blood combined with haploidentical transplantation scheme is assessed by comparing it with sibling fully matched transplantation using a non-inferiority analysis method (with the time of neutrophil engraftment after transplantation as the primary efficacy indicator). At the same time, indicators such as the time of platelet engraftment, infection incidence (including CMV, EBV incidence), acute/chronic GvHD incidence, transplant-related mortality (TRM), event-free survival (EFS), and overall survival (OS) are monitored to evaluate the safety and effectiveness of this scheme. |
Who may qualify
- Ages
- 6 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- A patient age of 6-75 years
- Patients diagnosed with Severe Aplastic Anemia (SAA) , Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) or Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT
- Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study
Key exclusion criteria (2 listed)
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
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 |
|---|---|---|
| Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.Conditioning regimen consisted of the following1)Severe aplastic anemia(SAA):FC-ATG:Flu 30 mg/m^2/d ×4d (- 7d- -4d), CTX 22.5 -25mg/kg/d × 4d (-7d- -4d), ATG 7.5mg in total（- 5d- -2d).2)Transfusion-dependent non-severe aplastic anemia（TD-NSAA) and Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA):BU/FC-ATG:Bu 0.8mg/kg(q6h -8d）+FC-ATG. The GVHD prophylaxis program was Application of Anti-CD25 Humanized Monoclonal Antibody 50mg (+1d,+4d);cyclosporine A + mycophenolate mofetil (MMF)+short course methotrexate (MTX).The infusion time of umbilical cord blood (UCB) stem cells in the UCB-assisted haploidentical hematopoietic stem cell transplantation (UCB-Haplo-HSCT) group: 4 hours before the infusion of peripheral blood stem cells.In the follow-up phase, outcome evaluation indicators must be collected according to the follow-up plan, ensuring data quality. | Procedure or surgery | Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study |
Primary outcome
Time to neutrophil engraftment
Time frame: 60 days
Neutrophil engraftment was defined as the first of three consecutive days on which the patient's absolute neutrophil count was 500/uL.
6 secondary outcomes
Time to platelet engraftment
Time frame: 60 days
Incidence of infections (including CMV and EBV incidence)
Time frame: 100 days,6 months,12 months,24 months
Overall survival rate (OS)
Time frame: 24 months
Event-free survival (EFS)
Time frame: 24 months
Incidence of acute/chronic GvHD
Time frame: 24 months
Transplant-related mortality (TRM)
Time frame: 100 days,6 months,12 months,24 months
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 14 Aug 2025.
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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.
Tao Wang, Dr. study director
Shanxi Bethune Hospital Regulatory Authority
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group. in people with Aplastic Anemias, run by Shanxi Bethune 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 3 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 14 Aug 2025. 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.
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 Tao Wang, Dr. (13835175119, wangtao99699@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to neutrophil engraftment (time frame: 60 days). 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 14 Aug 2025.