PNH drug safety study ends early: what happened?

NCT ID NCT05741346

First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 24 times

Summary

This study aimed to monitor the long-term safety of BCX9930 in people with paroxysmal nocturnal hemoglobinuria (PNH) who had already benefited from the drug in a previous study. It included 28 participants who continued taking BCX9930 because they had no other good treatment options. The study was terminated early, so results are limited.

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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

Conditions

Explore the condition pages connected to this study.