PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) explained in plain language.
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New drug pegcetacoplan tested in teens with rare blood disorder
Disease control Recruiting nowThis study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE2 • Sponsor: Apellis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 28, 2026 00:00 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 Jun 27, 2026 14:00 UTC
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New stem cell transplant trial offers 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 (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New siRNA drug ADX-038 enters human trials for PNH anemia
Disease control Recruiting nowThis study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1, PHASE2 • Sponsor: ADARx Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Hope for kids with rare blood disease: new pill under study
Disease control Recruiting nowThis study tests a medicine called iptacopan in children aged 2 to under 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. About 12 children will take the medicine, and doctors will monitor side effects and blood…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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New hope for rare blood and kidney diseases: iptacopan access program opens
Disease control AVAILABLEThis program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exha…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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New drug iptacopan tested in Real-Life PNH patients
Knowledge-focused Recruiting nowThis study looks at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:03 UTC
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New study tracks safety of ultomiris in pregnant women
Knowledge-focused Recruiting nowThis study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:07 UTC
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New pill CMS-D017 enters first human safety trials
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 conditions: paroxysmal nocturnal hemoglobinuria (a blood disorder) and complement-mediated kidney disease. Par…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1 • Sponsor: Shenzhen Kangzhe Biotechnology Co., Ltd. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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Thousands with blood diseases to be tracked in massive chinese registry
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 Jun 27, 2026 09:04 UTC
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Liver function may affect how PNH drug works
Knowledge-focused Recruiting nowThis early-stage study is testing a single dose of the drug HSK39297 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have mild or moderate liver impairment, compared to those with normal liver function. The goal is to see how the liver affects drug levels and safe…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Phase: PHASE1 • Sponsor: Haisco Pharmaceutical Group Co., Ltd. • Aim: Knowledge-focused
Last updated Jun 26, 2026 13:21 UTC