New pill shows promise for rare blood disorder in Long-Term trial
NCT ID NCT07177872
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took another drug (eculizumab) to see if NTQ5082 safely controls the disease long-term. The main goal is to check for side effects and whether it helps maintain healthy hemoglobin levels without needing 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, this could provide a new long-term treatment option to control PNH, reducing symptoms like fatigue and the need for blood transfusions.
What could go wrong
This is an open-label study with no placebo, so results may be biased. Long-term safety and effectiveness are still being evaluated, and the drug may not work for everyone.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences ,
Tianjin, Tianjin Municipality, 300020, China
Contact
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