New oral drug HRS-5965 shows promise for tough PNH anemia cases
NCT ID NCT06715943
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tested a new drug called HRS-5965 in 39 adults with paroxysmal nocturnal hemoglobinuria (PNH) who remained anemic even after standard anti-C5 antibody treatment. The study aimed to see if the drug could raise hemoglobin levels without needing blood transfusions. Participants took HRS-5965 capsules for 24 weeks, and researchers measured how many achieved stable hemoglobin levels without transfusions.
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, this could offer a new oral treatment option for PNH patients who still have anemia even after standard anti-C5 antibody therapy.
- What could go wrong
- This is a small, single-arm trial with only 39 participants, so results may not apply broadly. The drug's safety and effectiveness need confirmation in larger studies.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA are added.
By submitting, you agree to our Terms of use
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
-
Hematology Hospital of Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 30000, China
-
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, 30000, 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