New hope for PNH patients: early access to danicopan combo therapy

NCT ID NCT05982938

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, and have failed or cannot use other approved treatments. The goal is to control the disease by reducing red blood cell destruction outside blood vessels.

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Conditions

The condition(s) this trial relates to.

anemia Hemoglobinuria, Paroxysmal Hemolysis hemolytic anemia paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Research Site

    AVAILABLE

    Bologna, 40138, Italy

  • Research Site

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    Cagliari, 09121, Italy

  • Research Site

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    Catania, 95123, Italy

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    Civitanova Marche, 62012, Italy

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    Cremona, 26100, Italy

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    Cuneo, 12100, Italy

  • Research Site

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    Frosinone, 03100, Italy

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    Novara, 28100, Italy

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    Palermo, 90146, Italy

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    Pescara, 65100, Italy

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    Ravenna, 48121, Italy

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    Reggio Calabria, 89100, Italy

  • Research Site

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    Roma, 00161, Italy

  • Research Site

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    Roma, 00168, Italy

  • Research Site

    AVAILABLE

    Torino, 10126, Italy