New drug cocktail aims to free aplastic anemia patients from transfusions

NCT ID NCT07558434

Not yet recruiting Disease control Sponsor: Peking Union Medical College Hospital Source: ClinicalTrials.gov ↗

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.

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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

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