Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2026-06-18Clinical Trial Update: A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH [Phase 3]
Phase 3 long-term safety and efficacy study of danicopan added to a C5 inhibitor in paroxysmal nocturnal hemoglobinuria (PNH) is ongoing; st…
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CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Results: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 trial assessing efficacy, safety, PK/PD, and immunogenicity of Ravulizumab in Chinese adults with Paroxysmal Nocturnal Hemoglobinuri…
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CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Update: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 clinical trial update assessing ravulizumab in Chinese adults with paroxysmal nocturnal hemoglobinuria (PNH), reporting efficacy, sa…
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CLINICAL_TRIALS trial_result · 2026-06-03Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Phase 3 long-term safety and efficacy extension study for pegcetacoplan in PNH is active/not recruiting, continuing evaluation of extended o…
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CLINICAL_TRIALS trial_result · 2026-04-28Clinical Trial Update: A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors [Phase 3]
A Phase 3 trial comparing crovalimab to eculizumab in patients with paroxysmal nocturnal hemoglobinuria who are already treated with complem…
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CLINICAL_TRIALS trial_result · 2026-04-17Clinical Trial Update: Danicopan PMS in Korea
AstraZeneca’s Danicopan is enrolling in a post-marketing surveillance study in Korea for Paroxysmal Nocturnal Hemoglobinuria.
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH [Phase 3]
Phase 3 long-term safety and efficacy study evaluating danicopan as add-on therapy to C5 inhibitors for Paroxysmal nocturnal hemoglobinuria;…
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
An ongoing Phase 3 trial is evaluating the long-term safety and tolerability of iptacopan in patients with paroxysmal nocturnal hemoglobinur…
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris [Phase 2]
A Phase 2 study of NM8074 in adults with paroxysmal nocturnal hemoglobinuria (inadequate response to Soliris) is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice
This trial update reports the terminated evaluation of iptacopan for efficacy and safety in adults with paroxysmal nocturnal hemoglobinuria …
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CLINICAL_TRIALS trial_result · 2026-03-09Clinical Trial Update: Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) [Phase 2]
This update reports the completion of a Phase 2 study evaluating a treosulfan-based conditioning regimen before bone marrow or blood stem ce…
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CLINICAL_TRIALS trial_result · 2026-01-21Clinical Trial Update: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
This Phase 3 trial update assesses the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese ad…
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CLINICAL_TRIALS trial_result · 2026-01-08Clinical Trial Update: Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment [Phase 3]
Phase 3 clinical trial evaluating the efficacy and safety of HRS-5965 in Paroxysmal nocturnal hemoglobinuria patients who remain anemic afte…
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects [Phase 1]
Phase 1 trial comparing capsule and tablet formulations of HRS-5965 in healthy subjects has been completed.
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
This clinical trial update reports on the long-term safety and tolerability evaluation of HRS-5965 in patients with Paroxysmal Nocturnal Hem…
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CLINICAL_TRIALS trial_result · 2025-12-23Clinical Trial Update: Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis [Phase 3]
Phase 3 clinical trial update indicates danicopan as an add-on to ravulizumab or eculizumab in pediatric patients with paroxysmal nocturnal …
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CLINICAL_TRIALS trial_result · 2025-12-22Clinical Trial Update: A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy [Phase 3]
Phase III study evaluating HSK39297 in paroxysmal nocturnal hemoglobinuria patients naive to complement inhibitor therapy has completed. The…
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CLINICAL_TRIALS trial_result · 2025-12-18Clinical Trial Update: A Real-world, Multi-center, Prospective, Observational Study for PNH in China
A real-world, multi-center, prospective observational study on paroxysmal nocturnal hemoglobinuria (PNH) conducted in China has completed, s…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. [Phase 3]
Phase 3 single-arm, open-label trial evaluating switching from anti-C5 therapy to iptacopan for 24 weeks in patients with paroxysmal nocturn…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants [Phase 1]
Longbio Pharma has completed a Phase 1 trial evaluating single and multiple doses of LP-005 in healthy adult participants to explore its saf…
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CLINICAL_TRIALS trial_result · 2025-11-10Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors [Phase 3]
This Phase 3 trial compares crovalimab against eculizumab in adults with paroxysmal nocturnal hemoglobinuria who have not previously receive…
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CLINICAL_TRIALS trial_result · 2025-11-10Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors [Phase 3]
Phase 3 trial comparing crovalimab to eculizumab in treatment-naïve PNH patients; study status active/not recruiting with results posted in …
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CLINICAL_TRIALS trial_result · 2025-10-24Clinical Trial Update: Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
Phase 2 long-term safety update for BCX9930 in adults with paroxysmal nocturnal hemoglobinuria; the study is terminated and results have bee…
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CLINICAL_TRIALS trial_result · 2025-10-01Clinical Trial Update: Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab [Phase 2]
Phase 2 trial evaluating OMS906 in paroxysmal nocturnal hemoglobinuria patients with suboptimal response to ravulizumab reports safety and e…
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy [Phase 3]
A Phase 3 clinical trial update evaluating the efficacy and safety of oral HRS-5965 in adults with paroxysmal nocturnal hemoglobinuria who h…