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New drug cocktail aims to free aplastic anemia patients from transfusions

NCT ID NCT07558434

Disease control Sponsor: Peking Union Medical College Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests a combination of three drugs—cyclosporine, a thrombopoietin receptor agonist (eltrombopag or hetrombopag), and romiplostim—in 43 adults newly diagnosed with severe aplastic anemia or transfusion-dependent non-severe aplastic anemia. The goal is to see if this triple therapy can improve blood cell counts and help patients become independent of blood transfusions. Participants must not have a suitable donor for a stem cell transplant or be unable to receive standard treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
cyclosporine, eltrombopag or hetrombopag, and romiplostim
What this could lead to
If this works, it could offer a new drug-only option for people with severe aplastic anemia who cannot have a stem cell transplant, reducing their need for blood transfusions.
What could go wrong
This is a small, early-phase trial with only 43 participants, so results may not apply to everyone. The drugs can have side effects like kidney problems or infections, and the combination may not work better than existing treatments.

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.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 43 people

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

Expected to start

Apr 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.

Ages

18 years and older

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: 1. Age ≥ 18 years old; 2. Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet \< 30×10\^9/L; 3. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT); 4. Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes; 5. With baseline liver and kidney functions \<2 ULN; 6. ECOG score ≤ 2; 7. Signed the informed consent; Exclusion Criteria: 1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.; 2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics; 3. PNH clone ≥ 50%; 4. Received HSCT before enrollment; 5. Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs); 6. Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA; 7. Pregnant or lactating patients; 8. Severe bleeding or infection that cannot be controlled by standard treatment; 9. History of arterial or venous thrombosis; 10. Complicated with malignant tumors; 11. Participated in other clinical trials within 3 months; 12. Patients considered not suitable to participate in this study by the investigator.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Severe aplastic anemia (SAA) Transfusion-dependent non-severe aplastic anemia

How to take part

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  1. 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 ↗

  2. A doctor treating you

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More trials for these conditions

Other studies related to the condition(s) this trial covers.