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New hope for older adults with rare blood disorder: safer transplant regimen under study

NCT ID NCT07586735

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

About 64 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

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

40 to 60 years

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Peking Universtiy Peoples' Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100044, China

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