New drug combo aims to restore blood cells in severe anemia

NCT ID NCT07297550

Not yet recruiting Disease control Sponsor: Institute of Hematology & Blood Diseases Hospital, China Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called golidocitinib, combined with strong immune-suppressing therapy, for people with severe aplastic anemia—a condition where the bone marrow stops making enough blood cells. The trial has two parts: first, it checks safety in patients who didn't improve with standard treatment; then, it tests how well the drug works in newly diagnosed patients. Up to 42 participants will take golidocitinib capsules daily or every other day, and doctors will monitor side effects and blood cell recovery.

What this could mean

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

Active substance: Golidocitinib (a JAK1 inhibitor drug taken as capsules)
What this could lead to: If successful, this could offer a new treatment option for severe aplastic anemia, helping patients recover healthy blood cell counts.
What could go wrong: This is an early-phase trial with only 42 participants, so results may not apply broadly. The drug may cause side effects or fail to improve blood counts.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

NEWLY DIAGNOSED APLASTIC ANEMIA REFRACTORY APLASTIC ANEMIA SEVERE APLASTIC ANEMIA

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