New pill shows promise for rare blood disorder PNH in early trial

NCT ID NCT06764303

First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 25 times

Summary

This Phase 2 trial tests an experimental oral drug called NTQ5082 for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break down. The study involves 24 adults who have not previously received complement inhibitor therapy. The main goal is to see if the drug can raise hemoglobin levels by at least 20 g/L and reduce the need for blood transfusions.

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Contacts and locations

Locations

  • Hematology Hospital of the Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 250033, China

  • The First Affiliated Hospital, College of Medicine, Zhejiang University

    Hangzhou, Zhejiang, 310012, China

Conditions

Explore the condition pages connected to this study.