Severe Aplastic AnemiaNCT07586735
A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA
An observational study of Severe Aplastic Anemia and Aplastic Anaemia, run by Peking University People's Hospital at 1 site in China.
Trial summary
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT. Read more about this study
- Who is it for?
- Ages 40 to 60 years
- Conditions the study is for
- Severe Aplastic Anemia, Aplastic Anaemia, Hematopoietic Cell Transplant
- What is being tested?
- BFCA conditiong regimen
- How many people
- 64 participants
- Where
- 1 siteChina
- Study dates
- Jun 2026to 30 Dec 2029
- How long will I take part?
- 2 Years
- Phase
- Observational
- 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 40 to 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: Patients aged 40-60 years old who are diagnosed with severe aplastic anemia according to NCCN guidelines and who will receive haploidentical hematopoietic stem cell transplantation at the centers will be recruited.
Who may be able to join
- Severe aplastic anemia;
- Aged 40-60 years old;
- Weight 45Kg-100Kg;
- Eastern Cooperative Oncology Group (ECOG) score ≤3;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
- No severe infection;
- Subjects voluntarily participated in this clinical trial and signed the informed consent.
Who cannot join
- With other hematologic diseases;
- Expected survival of less than 1 month;
- Previous autologous or allogeneic hematopoietic stem cell transplantation;
- Pregnant patients;
- Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
- Other conditions that the investigator determines to be inappropriate for enrollment.
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- Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
- A history of symptomatic herpes zoster infection within 12 weeks prior to screening
- Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
- Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB
- Exposure to a live vaccine within 12 weeks prior to enrollment or expected to receive a live vaccine during the study
- Clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled
- Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure
- A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data
- Any of the following specific abnormalities on screening laboratory tests:
- ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN
- hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL)
- eGFR <50 mL/min/1.73 m2
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
Peking Universtiy Peoples' Hospital
Beijing, Beijing Municipality, 100044
Tingting Han, Doctor, 86-01088424953, htt1984.love@163.com
About this study
Official title: A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
- Being tested
- BFCA conditiong regimen
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BFCA conditiong regimenDrug
busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
| Study group | Role | What they receive |
|---|---|---|
| Peking unviversity Peoples' Hospital | Not described Receives: Drug: BFCA conditiong regimen | |
| The First Affiliated Hospital of Soochow University | Not described Receives: Drug: BFCA conditiong regimen | |
| Guangzhou First People's Hospital, | Not described Receives: Drug: BFCA conditiong regimen | |
| The First Affiliated Hospital of USTC | Not described Receives: Drug: BFCA conditiong regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Faliure free survival (FFS)
survival with a response to therapy after HSCT. Death, graft faliue and relapse were considered as treatment failure.
Measured: From enrollment to 2 years post-HSCT
Secondary outcomes (7)
The probability of Overall survival
Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up
Measured: From enrollment to 2 years post-HSCT
Myeloid and platelet engraftment
Myeloid and platelet engraftment were defined as international criteria.
Measured: 100 days post HSCT
The incidence of mixed chimerism
The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells.
Measured: 1 year post HSCT
The incidence of graft versus host disease(GVHD)
The severity of acute and chronic GVHD was evaluated according to standard criteria.
Measured: 100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD
Regimen related toxicity
The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988).
Measured: 100 days post HSCT
The incidence of Cytomegalovirus(CMV) and Epstein-Barr virus(EBV) reactivation
The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia.
Measured: 180 days post HSCT
The incidence of Transplantation related mortality
Transplantation related mortality was defined as death without disease progression.
Measured: 2 years post HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study startJun 2026 (estimated)
- Primary completionDec 2027 (estimated)
- Study completion30 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking University People's 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 64 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07586735
- Sponsor's protocol ID
- PUPH20260508
- Status last verified
- Jun 2026
- First submitted
- 8 May 2026
- Last updated
- 6 Jul 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 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial
- ClinicalTrials.gov ID
- NCT07586735
- Acronym / sponsor ID
- PUPH20260508
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- 30 Dec 2029 (estimated)
- First posted
- 14 May 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.
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Peking unviversity Peoples' Hospital | – | Drug: BFCA conditiong regimen |
| The First Affiliated Hospital of Soochow University | – | Drug: BFCA conditiong regimen |
| Guangzhou First People's Hospital, | – | Drug: BFCA conditiong regimen |
| The First Affiliated Hospital of USTC | – | Drug: BFCA conditiong regimen |
Who may qualify
- Ages
- 40 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Patients aged 40-60 years old who are diagnosed with severe aplastic anemia according to NCCN guidelines and who will receive haploidentical hematopoietic stem cell transplantation at the centers will be recruited.
Key inclusion criteria (7 listed)
- Severe aplastic anemia;
- Aged 40-60 years old;
- Weight 45Kg-100Kg;
- Eastern Cooperative Oncology Group (ECOG) score ≤3;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
Key exclusion criteria (18 listed)
- With other hematologic diseases;
- Expected survival of less than 1 month;
- Previous autologous or allogeneic hematopoietic stem cell transplantation;
- Pregnant patients;
- Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
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 |
|---|---|---|
| BFCA conditiong regimenbusulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2). | Drug | Guangzhou First People's Hospital,, Peking unviversity Peoples' Hospital, The First Affiliated Hospital of Soochow University, The First Affiliated Hospital of USTC |
Primary outcome
Faliure free survival (FFS)
Time frame: From enrollment to 2 years post-HSCT
survival with a response to therapy after HSCT. Death, graft faliue and relapse were considered as treatment failure.
7 secondary outcomes
The probability of Overall survival
Time frame: From enrollment to 2 years post-HSCT
Myeloid and platelet engraftment
Time frame: 100 days post HSCT
The incidence of mixed chimerism
Time frame: 1 year post HSCT
The incidence of graft versus host disease(GVHD)
Time frame: 100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD
Regimen related toxicity
Time frame: 100 days post HSCT
The incidence of Cytomegalovirus(CMV) and Epstein-Barr virus(EBV) reactivation
Time frame: 180 days post HSCT
The incidence of Transplantation related mortality
Time frame: 2 years post 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 6 Jul 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?
An observational study of Severe Aplastic Anemia and Aplastic Anaemia, run by Peking University People's 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 7 inclusion and 18 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 6 Jul 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.
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 Tingting Han (8601088326666, htt1984.love@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Faliure free survival (FFS) (time frame: From enrollment to 2 years post-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 6 Jul 2026; AzTrials last refreshed its copy on 7 Nov 2026.