BCX9930 safety checked in PNH patients who benefited from treatment

NCT ID NCT05741346

Terminated Disease control Sponsor: BioCryst Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

Locations

Investigative Site

Paris, France
Investigative Site

Budapest, Hungary
Investigative Site

Ampang, Malaysia
Investigative Site

Bloemfontein, South Africa
Investigative Site

Cape Town, South Africa
Investigative Site

Pretoria, South Africa
Investigative Site

Daejeon, South Korea
Investigative Site

Barcelona, Spain
Investigative Site

Valencia, Spain
Investigative Site

Leeds, United Kingdom
Investigative Site

London, United Kingdom

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