PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Clinical trials for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA explained in plain language.
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Oral pill shows promise for rare blood disease, could replace injections
Disease control CompletedThis study tested whether adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, could safely switch from their standard injection treatment to a new oral medication called iptacopan. Fifty-two patients who were already doing well on their current treatment…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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New hope for PNH patients who still struggle with anemia despite current treatments
Disease control CompletedThis Phase 3 trial tested whether HRS-5965 capsules could help patients with paroxysmal nocturnal hemoglobinuria (PNH) who continue to have anemia despite already receiving standard C5 inhibitor treatments like eulizumab. The study involved 39 adults who took the capsules for 24 …
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Chengdu Suncadia Medicine Co., Ltd. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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New oral treatment challenges standard therapy for rare blood disorder
Disease control CompletedThis study tested whether HRS-5965 capsules work as well as the standard treatment (eculizumab) for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The 24-week trial involved 76 patients who had not previously received complement inhibitor therapy. Researchers m…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Chengdu Suncadia Medicine Co., Ltd. • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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New drug shows promise in controlling rare blood disorder
Disease control CompletedThis completed Phase 3 study tested whether the drug ravulizumab could effectively control paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease, in Chinese adults who hadn't previously tried this type of medication. The trial involved 18 participants and me…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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New transplant method aims to tame dangerous side effects for rare blood disease patients
Disease control CompletedThis study tested a new combination of drugs given before a bone marrow or blood stem cell transplant. The goal was to see if this new 'conditioning' regimen could help patients with rare bone marrow failure diseases have fewer and less severe complications after their transplant…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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New drug tested for rare blood disorder when standard treatment fails
Disease control CompletedThis study tested a new drug called OMS906 for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. It specifically looked at patients who weren't getting good results from their current standard treatment, ravulizumab. Researchers gave 12 participants two…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Phase: PHASE2 • Sponsor: Omeros Corporation • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Landmark study maps rare blood disease in china
Knowledge-focused CompletedThis study aimed to better understand a rare and serious blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH) in China. Researchers observed 724 patients with PNH to learn about their disease characteristics, symptoms, and how different treatments, including a newer dru…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:16 UTC
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Massive study tracks rare blood disease in over 6,000 patients
Knowledge-focused CompletedThis registry study collected information from over 6,000 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The main goal was to monitor long-term safety of specific treatments and understand how the disease progresses over time. Researchers gathered…
Matched conditions: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Feb 23, 2026 14:52 UTC