Aplastic AnaemiaNCT07338422
HID-HSCT Versus IST as First-line Treatment for SAA
A study testing IST (ATG + CsA+TPORA) and tranplantation in people with Aplastic Anaemia, run by Institute of Hematology & Blood Diseases Hospital, China.
Trial summary
This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Aplastic Anaemia
- What is being tested?
- IST (ATG + CsA+TPORA) and tranplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 116 participants
- Where
- No sites listed yet
- Study dates
- 14 Jan 2026to 14 Sep 2030
- 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 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- (1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.
- Age 14-70 years. (3) No HLA-matched related donor available. (4) HIV negative, HBV negative, HCV negative. (5) No absolute contraindications to transplantation or immunosuppressive therapy.
- Signed informed consent form must be obtained before study procedures begin; for subjects aged 18 years or older, informed consent shall be signed by the patient themselves or direct family members. Considering the patient's medical condition, if the patient's own signature is medically inadvisable, the informed consent shall be signed by a legal guardian or the patient's direct family member.
Who cannot join
- Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;
- Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;
- Known severe allergy to ATG;
- Previous allogeneic or autologous hematopoietic stem cell transplantation;
- Previous solid organ transplantation;
- Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;
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- Active HIV replication at enrollment, detectable HCV antibody positivity and HCV-RNA positivity within 90 days prior to enrollment, or HBsAg positivity; known seropositivity for HIV or active hepatitis C virus;
- History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);
- Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;
- Inability or unwillingness to sign the consent form;
- Other special circumstances deemed ineligible by the investigator.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia
This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
- Conditions
- Aplastic Anaemia
- Being tested
- IST (ATG + CsA+TPORA), tranplantation
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IST (ATG + CsA+TPORA)Procedure or surgery
The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA).
tranplantationProcedure or surgery
The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
Patients in the transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).
| Study group | Role | What they receive |
|---|---|---|
| HID-HSCT | Experimental | Not described Receives: Procedure: tranplantation |
| IST | Other | Not described Receives: Procedure: IST (ATG + CsA+TPORA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Failure Free Survival, FFS
survival with complete response whereas death, graft failure and relapse are considered treatment failures
Measured: 2 year
Secondary outcomes (6)
Overall Survival, OS
Measured: 2 year
Treatment Related Mortality, TRM
Transplantation-related mortality (TRM) was defined as death without graft failure
Measured: 2 year
hematology remission
hematology recovery, including WBC, Hb and PLT
Measured: 2 year
Infection incidence
Measured: 2 year
GVHD incidence
Measured: 2 year
QoL evaluation
life quality assessment
Measured: 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2026
- Study start14 Jan 2026 (estimated)
- Primary completion14 Sep 2030 (estimated)
- Study completion14 Sep 2030 (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 Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 116 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07338422
- Sponsor's protocol ID
- IIT2025089
- Status last verified
- Nov 2025
- First submitted
- 25 Sep 2025
- Last updated
- 13 Jan 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 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07338422
- Acronym / sponsor ID
- IIT2025089
- Conditions
- Aplastic Anaemia
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 116 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 14 Jan 2026 (estimated)
- Primary completion
- 14 Sep 2030 (estimated)
- Study completion
- 14 Sep 2030 (estimated)
- First posted
- 13 Jan 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.
This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HID-HSCT | Experimental | Procedure: tranplantation |
| IST | Other | Procedure: IST (ATG + CsA+TPORA) |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- (1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.
Key exclusion criteria (11 listed)
- Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;
- Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;
- Known severe allergy to ATG;
- Previous allogeneic or autologous hematopoietic stem cell transplantation;
- Previous solid organ transplantation;
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 |
|---|---|---|
| IST (ATG + CsA+TPORA)The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation. Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA). | Procedure or surgery | IST |
| tranplantationThe selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation. Patients in the transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT). | Procedure or surgery | HID-HSCT |
Primary outcome
Failure Free Survival, FFS
Time frame: 2 year
survival with complete response whereas death, graft failure and relapse are considered treatment failures
6 secondary outcomes
Overall Survival, OS
Time frame: 2 year
Treatment Related Mortality, TRM
Time frame: 2 year
hematology remission
Time frame: 2 year
Infection incidence
Time frame: 2 year
GVHD incidence
Time frame: 2 year
QoL evaluation
Time frame: 2 year
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 13 Jan 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.
Questions about this trial
What is this clinical trial studying?
A study testing IST (ATG + CsA+TPORA) and tranplantation in people with Aplastic Anaemia, run by Institute of Hematology & Blood Diseases Hospital, 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 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 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Xiaoyu Zhang (022-23608569, zhangxiaoyu@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Failure Free Survival, FFS (time frame: 2 year). 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 13 Jan 2026.