New hope for rare blood disorder: ravulizumab trial shows promise in china
NCT ID NCT06578949
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This completed Phase 3 trial tested ravulizumab in 18 adults in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study measured changes in LDH, a marker of red blood cell breakdown, to see if the drug could control the disease. All participants had not previously received complement inhibitor treatment.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ravulizumab
- What this could lead to
- If successful, ravulizumab could offer a new treatment option for people with PNH in China, helping control red blood cell destruction and reduce symptoms.
- What could go wrong
- This is a small, single-arm study with only 18 participants, so results may not apply broadly. Ravulizumab requires lifelong treatment and carries infection risks, including meningococcal infection.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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18 people
The number who actually took part.
- Started
-
Oct 2024
- Finished
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Dec 2025
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adult C5 inhibitor naive PNH patients (age\>=18), which is confirmed by flow cytometry evaluation. * Must be vaccinated againast N meningitidis. Exclusion Criteria * Meningitidis infection or unresolved meningococcal disease * History of bone marrow transplantation * Other significant systemic diseases that might have impact on efficacy and safety assessment
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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
-
Research Site
Beijing, CN-100730, China
-
Research Site
Guangzhou, 510100, China
-
Research Site
Hangzhou, 310003, China
-
Research Site
Nantong, 226001, China
-
Research Site
Shanghai, 200040, China
-
Research Site
Tianjin, 300020, China
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Research Site
Tianjin, 300050, China
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Research Site
Wuhan, 430022, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Hidden blood disorder may explain mysterious strokes in young adults
- 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