PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) explained in plain language.
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Half-Matched stem cell transplant offers new hope for rare blood diseases
Disease control Recruiting nowThis study tests a stem cell transplant using blood stem cells from a half-matched family donor (like a parent or sibling) for people with severe aplastic anemia, a related bone marrow disorder, or a rare blood disease called PNH. Participants receive chemotherapy and a one-time …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated May 17, 2026 00:44 UTC
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New drug ADX-038 aims to control anemia in PNH patients
Disease control Recruiting nowThis study tests a new drug called ADX-038 for people with PNH who still have anemia despite treatment. It first checks safety in healthy adults, then tests the drug in PNH patients. The goal is to see if ADX-038 can safely control the disease and improve red blood cell health.
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1, PHASE2 • Sponsor: ADARx Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 00:32 UTC
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Hope for kids with rare blood disorder: new pill under study
Disease control Recruiting nowThis study tests a medicine called iptacopan in 12 children aged 2 to 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. PNH causes red blood cells to break apart, and this treatment aims to control that damage.
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New hope for PNH patients with stubborn anemia?
Disease control Recruiting nowThis study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1, PHASE2 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New hope for rare blood and kidney diseases: early access to iptacopan
Disease control AVAILABLEThis program offers early access to the drug iptacopan for people with serious conditions like C3 glomerulopathy (a kidney disease) or paroxysmal nocturnal hemoglobinuria (a blood disorder) who have no other treatment options. Doctors can request the drug for patients who are not…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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New PNH drug iptacopan tested in everyday patients
Disease control Recruiting nowThis study looks at how well the drug iptacopan works for people with a rare blood disease called PNH. Researchers will track 50 patients in Italy for 12 months to see if their blood health improves and if they need fewer blood transfusions. The goal is to understand the drug's r…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 04, 2026 16:30 UTC
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New drug CMS-D017 takes first steps in human testing
Knowledge-focused Recruiting nowThis early-stage study tests a new drug called CMS-D017 in 88 healthy adults to see if it is safe and how the body processes it. The drug is being developed for two rare blood and kidney diseases. Participants receive either the drug or a placebo, and researchers monitor side eff…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1 • Sponsor: Shenzhen Kangzhe Biotechnology Co., Ltd. • Aim: Knowledge-focused
Last updated May 17, 2026 00:46 UTC
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New study monitors ultomiris safety for pregnant women and their babies
Knowledge-focused Recruiting nowThis study looks at the safety of the drug Ultomiris when used during pregnancy. Researchers will track 75 pregnant or postpartum women who take Ultomiris for conditions like PNH or aHUS. They will record pregnancy complications, maternal health issues, and infant health outcomes…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 15, 2026 11:54 UTC
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Massive blood disease registry launches in china
Knowledge-focused Recruiting nowThis study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Institute of Hematology & Blood Diseases Hospital, China • Aim: Knowledge-focused
Last updated May 13, 2026 15:59 UTC
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Liver function may affect new PNH drug processing
Knowledge-focused Recruiting nowThis study looks at how the body processes a new drug called HSK39297 for paroxysmal nocturnal hemoglobinuria (PNH) in people with mild or moderate liver problems compared to those with healthy livers. About 24 adults will receive a single dose to measure drug levels and safety. …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1 • Sponsor: Haisco Pharmaceutical Group Co., Ltd. • Aim: Knowledge-focused
Last updated May 11, 2026 20:52 UTC