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PNH

Clinical trials for PNH explained in plain language.

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Trials to join now! 2 Not yet finished but already full! 2 Completed 2
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  • Researchers track Long-Term safety of rare blood disorder treatment

    Disease control Ongoing

    This study is observing the long-term safety of adding a medication called danicopan to standard treatments for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. It will follow about 50 patients already taking this combination therapy to monitor for ser…

    Matched conditions: PNH

    Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control

    Last updated Mar 30, 2026 14:35 UTC

  • Researchers track Long-Term safety of rare blood disorder treatment

    Disease control Ongoing

    This study aims to understand the long-term safety of adding the drug danicopan to standard treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. Researchers will analyze existing health data from about 50 adult patients already taking these medications. …

    Matched conditions: PNH

    Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control

    Last updated Mar 30, 2026 14:32 UTC

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