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
128 clinical trials for this condition and its sub-types.
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Broader categories
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PNH drug iptacopan tested in Real-World setting – but study cut short
Disease control TerminatedThis 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 Jun 27, 2026 12:06 UTC
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Can extra care after transplant help older patients live better?
Disease control TerminatedThis study tested two extra care programs—supportive palliative care and clinical management of other health issues—for older or frail patients who had a donor stem cell transplant. The goal was to see if these programs could improve quality of life and survival compared to stand…
Phase: PHASE2, PHASE3 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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BCX9930 safety checked in PNH patients who benefited from treatment
Disease control TerminatedThis 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: PHASE2 • Sponsor: BioCryst Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC