PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH explained in plain language.
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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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New hope for kids with rare blood disorder: first pediatric trial of oral drug begins
Disease control Recruiting nowThis study is testing an oral medication called iptacopan in children and teenagers (ages 2 to 18) with a rare and serious blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). The main goals are to see how the drug behaves in children's bodies and to check if it is saf…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 09, 2026 14:23 UTC
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First look: how a new blood disorder drug works in people with liver issues
Knowledge-focused Recruiting nowThis early-stage study aims to understand how a potential new drug for a rare blood disease (Paroxysmal Nocturnal Hemoglobinuria or PNH) is processed by the body in people with mild to moderate liver problems. It will compare the drug's levels and safety in 24 people who have liv…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH
Phase: PHASE1 • Sponsor: Haisco Pharmaceutical Group Co., Ltd. • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:15 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