PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA explained in plain language.
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New hope for kids with rare blood disease: Add-On pill may boost red blood cells
Disease control Recruiting nowThis study tests whether adding danicopan (a pill) to standard PNH therapy (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia due to extravascular hemolysis. About 6 participants…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 00:49 UTC
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New hope for PNH patients: experimental drug HS-10542 enters Mid-Stage trial
Disease control Recruiting nowThis study tests a new drug, HS-10542, in adults with paroxysmal nocturnal hemoglobinuria (PNH) who still have signs of active red blood cell destruction despite current treatments. About 50 participants will receive the drug to see if it safely raises hemoglobin levels and reduc…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE1, PHASE2 • Sponsor: Jiangsu Hansoh Pharmaceutical Co., Ltd. • Aim: Disease control
Last updated May 17, 2026 00:44 UTC
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Fabhalta under the microscope: new study tracks Real-World safety
Disease control Recruiting nowThis study watches 100 people with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Fabhalta. Researchers will track infections, side effects, and what happens if the drug is stopped. The goal is to see how safe the medicine is in everyday use over 48 weeks.
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 15, 2026 11:56 UTC
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New drug combo aims to control rare blood disease Long-Term
Disease control Recruiting nowThis study is for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It tests the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together. About 202 participants will receive the combination therapy to see …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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New drug iptacopan aims to improve life for PNH patients in china
Disease control Recruiting nowThis study tests how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes over time. About 80 participants in China who have not used similar t…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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New drug duo aims to tame blood disorder when standard meds fail
Disease control Recruiting nowThis study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose current C5 inhibitor therapy isn't fully controlling red blood cell destruction. The main goal is to see if the combination lowers a key b…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated May 11, 2026 20:52 UTC
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Korean PNH patients monitored on new add-on drug danicopan
Disease control Recruiting nowThis study is for people in Korea with a rare blood disease called PNH. It looks at how safe and effective the drug Danicopan is when added to their usual treatment. About 8 participants will be followed in regular clinic visits. The goal is to confirm the drug's known safety and…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: AstraZeneca • Aim: Disease control
Last updated May 11, 2026 20:40 UTC
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New hope for PNH patients: early access to danicopan combo therapy
Disease control AVAILABLEThis program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant anemia despite current treatment with Soliris or Ultomiris. Participants must be at least 18 years old, have been on Soliris or Ultomiris for 6 months, a…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 11, 2026 20:38 UTC