PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) explained in plain language.
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New oral drug tested in kids with rare blood disease
Disease control Recruiting nowThis study aims to understand how a drug called iptacopan works in the bodies of children and teenagers with PNH, a rare and serious blood disease. It will test the drug's safety and how well it is tolerated in about 12 participants aged 2 to 18 years old. The goal is to see if t…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Family donor transplant offers hope for tough blood diseases
Disease control Recruiting nowThis study is testing a stem cell transplant from a partially matched family member for people with severe aplastic anemia, myelodysplastic syndrome (MDS), or paroxysmal nocturnal hemoglobinuria (PNH). These are serious disorders where the bone marrow fails to make enough healthy…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Last-Hope drug access opens for devastating rare diseases
Disease control AVAILABLEThis program provides access to the investigational drug iptacopan for patients with two rare, serious conditions: C3 glomerulopathy (a kidney disease) and paroxysmal nocturnal hemoglobinuria (a blood disorder). It's for patients who have no other treatment options available and …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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New hope for PNH patients: trial tests Next-Gen drug for stubborn anemia
Disease control Recruiting nowThis study is testing an experimental drug called ALN-CFB in people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who still have anemia despite being on standard C5 inhibitor therapy. The main goals are to check the drug's safety, how the body processes it, and see if it can hel…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1, PHASE2 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC
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First look: how a new blood disorder drug behaves in damaged livers
Knowledge-focused Recruiting nowThis early-stage study aims to understand how a potential drug for a rare blood disease (PNH) is processed by the body in people with mild to moderate liver problems. It will compare drug levels and safety in 24 participants, some with liver impairment and some with normal liver …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1 • Sponsor: Haisco Pharmaceutical Group Co., Ltd. • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:41 UTC
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Massive study launches to map China's blood disease landscape
Knowledge-focused Recruiting nowThis study aims to gather detailed information on many different blood diseases in China, including cancers like leukemia and lymphoma, and conditions like hemophilia. It will follow 2,300 patients to learn how common these diseases are, what treatments patients receive, how well…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Institute of Hematology & Blood Diseases Hospital, China • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:14 UTC
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Tracking baby health: study follows pregnant women on rare disease drug
Knowledge-focused Recruiting nowThis study aims to gather information on the safety of the drug Ultomiris (ravulizumab) during pregnancy and breastfeeding. It will follow about 75 women who have rare conditions like PNH or aHUS and who took Ultomiris while pregnant. Researchers will track the health of both the…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Feb 23, 2026 14:57 UTC