New pill aims to tame rare blood disorder PNH

NCT ID NCT06764303

Ongoing Disease control Sponsor: Nanjing Chia-tai Tianqing Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This Phase 2 trial tests NTQ5082, an oral drug, in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart. The drug blocks a part of the immune system to prevent this destruction. The main goal is to see if it can raise hemoglobin levels and reduce the need for blood transfusions.

What this could mean

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

Active substance: NTQ5082 capsules
What this could lead to: If successful, NTQ5082 could offer a new oral treatment option for PNH, helping to control red blood cell destruction and reduce the need for blood transfusions.
What could go wrong: This is an early Phase 2 trial with only 24 participants, so results may not apply broadly. The drug may not work as hoped or could have side effects not yet seen.

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.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

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

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