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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100730, China
-
The Blood Disease Hospital of the Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A pill that could replace infusions for a rare blood disease?
- Every-8-Week shot could simplify PNH treatment
- Real-World test: can a targeted drug keep PNH in check for years?
- New transplant method aims to reduce complications in bone marrow failure patients
- No treatment, just observation: large study looks at PNH blood cells
- New hope for rare blood disorder: Long-Term study of XH-S003 underway