BCX9930 safety checked in PNH patients who benefited from treatment
NCT ID NCT05741346
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at the long-term safety of BCX9930, an oral medication, for people with paroxysmal nocturnal hemoglobinuria (PNH) who had already benefited from the drug in a previous study. It included 28 participants and aimed to monitor side effects. The study was terminated early, so results are limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
BCX9930 (oral medication)
What this could lead to
If successful, this could provide a long-term treatment option for people with PNH who have limited alternatives.
What could go wrong
The study was terminated early and only included 28 people, so results are limited. Long-term safety and effectiveness are not fully established.
Disclaimer
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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.
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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Investigative Site
Paris, France
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Investigative Site
Budapest, Hungary
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Investigative Site
Ampang, Malaysia
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Investigative Site
Bloemfontein, South Africa
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Investigative Site
Cape Town, South Africa
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Investigative Site
Pretoria, South Africa
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Investigative Site
Daejeon, South Korea
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Investigative Site
Barcelona, Spain
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Investigative Site
Valencia, Spain
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Investigative Site
Leeds, United Kingdom
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Investigative Site
London, United Kingdom