New pill could rival standard infusion for rare blood disorder
NCT ID NCT06593938
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This Phase 3 study tested a new oral medication, HRS-5965, against the standard infusion eculizumab in 76 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The main goal was to see if HRS-5965 could raise hemoglobin levels to normal without needing blood transfusions. Participants took the drug for 24 weeks, and researchers measured blood counts and transfusion needs.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
HRS-5965 capsule
What this could lead to
If successful, HRS-5965 could offer a new oral treatment option for PNH, potentially improving anemia and reducing the need for blood transfusions.
What could go wrong
This is a relatively small Phase 3 trial (76 participants) and results may not apply to all PNH patients. The drug is compared to an existing treatment, so benefits over standard care are uncertain.
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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Locations
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Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100730, China
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The Blood Disease Hospital of the Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020, China