PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA explained in plain language.
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Three-year trial tests long-term safety of new PNH combination therapy
Disease control OngoingThis study follows patients with a rare blood disease called PNH for up to three years. It tests the long-term safety and effectiveness of adding a drug called danicopan to their standard treatment. The goal is to see if this combination helps control the disease better over time…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:43 UTC
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Researchers track Long-Term safety of rare blood disorder treatment
Disease control OngoingThis study is observing the long-term safety of adding a medication called danicopan to standard treatments for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. It will follow about 50 patients already taking this combination therapy to monitor for ser…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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Rare blood disease patients get continued access to experimental treatment
Disease control NO_LONGER_AVAILABLEThis program provides continued access to an experimental two-drug combination (pozelimab and cemdisiran) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease where the immune system destroys red blood cells. It's specifically for patients who have already co…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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Researchers track Long-Term safety of rare blood disorder treatment
Disease control OngoingThis study aims to understand the long-term safety of adding the drug danicopan to standard treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. Researchers will analyze existing health data from about 50 adult patients already taking these medications. …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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New drug challenges standard treatment for rare blood disease
Disease control OngoingThis study is testing whether a new drug called crovalimab works as well as the current standard treatment (eculizumab) for controlling paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease. It involves 210 adults with PNH who have never taken this type of m…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 25, 2026 14:09 UTC
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New drug trial aims to control rare blood disease in untreated patients
Disease control OngoingThis study is testing whether the drug crovalimab can safely control paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease where the immune system destroys red blood cells. The trial includes about 50 people aged 12 and older with PNH who have never taken similar comple…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 23, 2026 15:17 UTC
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New capsule targets root cause of debilitating blood disease
Disease control OngoingThis study is testing an experimental capsule called NTQ5082 for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease. The trial aims to see if the treatment safely improves patients' red blood cell counts and reduces symptoms like severe fatigu…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE2 • Sponsor: Nanjing Chia-tai Tianqing Pharmaceutical • Aim: Disease control
Last updated Mar 18, 2026 18:25 UTC
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New drug's long-term safety tested for rare blood disease
Disease control OngoingThis study is checking the long-term safety and side effects of the drug HRS-5965 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disorder. It is open to 132 patients who have already taken HRS-5965 in a previous study and seemed to benefit from…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE2 • Sponsor: Chengdu Suncadia Medicine Co., Ltd. • Aim: Disease control
Last updated Mar 05, 2026 13:59 UTC
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New drug challenges standard treatment for rare blood disease
Disease control OngoingThis study is testing whether a new drug called crovalimab works as well as or better than the standard treatment (eculizumab) for controlling paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It will involve about 190 people who are already being treated for PNH.…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 02, 2026 15:26 UTC