PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA explained in plain language.
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New hope for PNH patients failing standard therapy
Disease control Recruiting nowThis study is testing a new combination of two drugs, pozelimab and cemdisiran, for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) whose current C5 inhibitor treatment isn't fully controlling the destruction of their red blood cells. The main goal is to see if this combina…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 21:26 UTC
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Oral pill trial offers new hope for controlling rare blood disease
Disease control Recruiting nowThis study is testing a new oral medication called HS-10542 for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease where the immune system destroys red blood cells. The goal is to see if the pill can safely control the disease and improve blood counts, especial…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE1, PHASE2 • Sponsor: Jiangsu Hansoh Pharmaceutical Co., Ltd. • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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New Two-Drug combo aims for Long-Term control of rare blood disease
Disease control Recruiting nowThis study is testing the long-term safety and effectiveness of an experimental two-drug treatment (pozelimab and cemdisiran) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease. The goal is to see if this combination can safely control the…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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New drug study aims to transform treatment for rare blood disorder
Disease control Recruiting nowThis study is observing how a drug called iptacopan affects the management and outcomes of a rare blood disease called paroxysmal nocturnal hemoglobinuria (PNH) in real-world medical settings in China. It will enroll 80 adult patients who are starting iptacopan treatment, either …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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New drug duo challenges standard PNH treatments in major trial
Disease control Recruiting nowThis Phase 3 study is testing whether a new combination of two drugs, pozelimab and cemdisiran, works better and is safer for people with paroxysmal nocturnal hemoglobinuria (PNH) compared to two existing standard treatments. The trial will enroll 190 adults with active PNH who h…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 14:41 UTC
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New hope for PNH patients: early access to additional treatment
Disease control AVAILABLEThis program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already receiving standard treatments but still experience significant red blood cell destruction. Participants must be on SOLIRIS or ULTOMIRIS for at least 6 months …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Apr 02, 2026 14:56 UTC
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Drug study aims to free PNH patients from blood transfusions
Disease control Recruiting nowThis study observes how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH) during routine medical care in Russia. Researchers will follow 100 patients already prescribed iptacopan by their doctors to see if it increases their hemoglobin levels…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Apr 02, 2026 14:56 UTC
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New Drug's Real-World safety under watch in blood disorder patients
Disease control Recruiting nowThis study is monitoring the safety and effectiveness of Fabhalta capsules in 100 patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track patients for 48 weeks after starting treatment to watch for infections, side effects, and how w…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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New hope for kids with rare blood disorder
Disease control Recruiting nowThis study is testing whether adding a new drug called danicopan helps children with a rare blood disease called PNH. The children are already on standard treatment but still have significant anemia. The main goal is to see if the add-on drug increases their hemoglobin levels (a …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC