New combo therapy offers hope for rare bone marrow failure

NCT ID NCT07297550

First seen Jan 11, 2026 · Last updated May 14, 2026 · Updated 21 times

Summary

This study tests a new drug (JAK1 inhibitor) combined with strong immune-suppressing therapy for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The trial has two parts: first, a small safety test in patients who didn't improve with standard treatment, then a larger test in newly diagnosed patients. The goal is to see if the combination is safe and helps restore normal blood cell counts.

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