Paroxysmal nocturnal hemoglobinuria
MONDO:0100244Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder characterized by corpuscular hemolytic anemia, bone marrow failure and frequent thrombotic events.
Also known as: Marchiafava-Micheli disease, PNH, acquired paroxysmal nocturnal hemoglobinuria, hereditary paroxysmal nocturnal hemoglobinuria, inherited paroxysmal nocturnal hemoglobinuria, paroxysmal hemoglobinuria
83 clinical trials for this condition and its sub-types, 73 tagged with Paroxysmal nocturnal hemoglobinuria itself.
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Sub-types of Paroxysmal nocturnal hemoglobinuria
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PNH drug iptacopan tested in Real-World setting – but study cut short
Disease control Stopped earlyThis study looked at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) when used in everyday medical practice in Russia. Only 6 adults took part, and the study was terminated early. Researchers measured whether patients' hemoglobin levels…
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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BCX9930 safety checked in PNH patients who benefited from treatment
Disease control Stopped earlyThis 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…
Phase 2 • Sponsor: BioCryst Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC