PNH
Clinical trials for PNH explained in plain language.
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New drug combo for rare blood disease under Long-Term safety watch
Disease control OngoingThis study looks at the long-term safety of adding danicopan to standard treatments (eculizumab or ravulizumab) for adults with PNH, a rare blood disorder. Researchers will track serious side effects and infections over time using registry data from 50 participants. The goal is t…
Matched conditions: PNH
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 22, 2026 14:03 UTC
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New study tracks Long-Term safety of danicopan combo for rare blood disorder
Disease control OngoingThis study monitors the long-term safety of danicopan when used alongside standard treatments (ravulizumab or eculizumab) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track side effects, infections, and other health issues in …
Matched conditions: PNH
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 22, 2026 13:52 UTC
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New hope for PNH patients: Long-Term drug safety trial underway
Disease control OngoingThis study looks at the long-term safety and effectiveness of pegcetacoplan in people with PNH, a rare blood disorder. Participants must have completed a prior pegcetacoplan study and shown benefit. The main goal is to track side effects over time.
Matched conditions: PNH
Phase: PHASE3 • Sponsor: Apellis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 12, 2026 13:41 UTC