New hope for older adults with rare blood disorder: safer transplant regimen under study
NCT ID NCT07586735
First seen Jun 24, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This study tests a new combination of drugs (busulfan, fludarabine, cyclophosphamide, and r-ATG) as preparation for a stem cell transplant in people aged 40-60 with severe aplastic anemia. The goal is to see if this regimen helps the transplant succeed with fewer failures. The study will follow 64 participants across multiple centers to measure survival and recovery.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- busulfan, fludarabine, cyclophosphamide, and rabbit anti-thymocyte globulin (r-ATG)
- What this could lead to
- If successful, this could offer a safer transplant option for older adults with severe aplastic anemia, potentially improving survival and reducing complications.
- What could go wrong
- This is an early-stage observational study with only 64 participants, so results may not apply to everyone. The drug combination can cause serious side effects like infection or organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 64 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jun 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
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.
- Ages
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40 to 60 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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. Exclusion Criteria: * 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. * 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: 1. ALT or AST \>2 x ULN, or total bilirubin ≥1.5 x ULN 2. 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) 3. eGFR \<50 mL/min/1.73 m2
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Peking Universtiy Peoples' Hospital
RECRUITINGBeijing, Beijing Municipality, 100044, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cell transplants tested as a cure path for older aplastic anemia patients
- Can a gentler chemo-radiation combo make stem cell transplants safer for blood cancers?
- Can a simple video and kit program help kids take their transplant meds?
- Half-Matched stem cell transplant offers hope for children with blood disorders
- New transplant approach aims to tame bone marrow failure
- Can training transplant doctors in palliative care ease Patients' suffering?