Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS research · 2026-06-05Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Phase 3 long-term safety and efficacy extension study for pegcetacoplan in paroxysmal nocturnal hemoglobinuria (PNH) completed.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently [Phase 3]
This is a Phase 3 clinical trial evaluating a combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis [Phase 2]
Phase 2 clinical trial evaluating haplo-identical transplantation with post-transplant cyclophosphamide for GVHD prophylaxis in severe aplas…
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients [Phase 1, Phase 2]
This document reports an update on the safety, tolerability, pharmacokinetics and pharmacodynamics of ADX-038 in healthy participants and Pa…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
A clinical trial update on the REAL-CARE study evaluating the real-world effectiveness of iptacopan in Italian patients with paroxysmal noct…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients [Phase 3]
Phase 3 study to assess the pharmacokinetics, safety, and tolerability of iptacopan in pediatric patients with paroxysmal nocturnal hemoglob…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
This clinical trial update indicates that a study evaluating the safety of Ravulizumab (Ultomiris) in pregnant and postpartum individuals is…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Specified Drug-use Surveillance of Fabhalta Capsules
Novartis is conducting a specified drug-use surveillance trial of Fabhalta Capsules in people with Paroxysmal nocturnal hemoglobinuria, curr…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 1, Phase 2]
A clinical trial update for HS-10542 in paroxysmal nocturnal hemoglobinuria, currently in Phase 1/2 and recruiting for safety and efficacy a…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants [Phase 1]
This is a Phase 1 clinical trial update evaluating CMS-D017 after single and multiple doses in healthy participants, and the study is curren…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Long-term safety and efficacy extension study of pegcetacoplan in paroxysmal nocturnal hemoglobinuria (PNH) is ongoing in Phase 3 and is cur…
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works [Phase 3]
Phase 3 study evaluating long-term safety and efficacy of Pozelimab plus Cemdisiran in adults with paroxysmal nocturnal hemoglobinuria; recr…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor [Phase 1, Phase 2]
Phase 1/2 first-in-human trial evaluating ALN-CFB, an siRNA against complement factor B, for adults with paroxysmal nocturnal hemoglobinuria…
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Danicopan Early Access Program
An early access program for danicopan is described for patients with paroxysmal nocturnal hemoglobinuria (PNH), including those with extrava…
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment [Phase 3]
Phase 3 clinical trial evaluating the safety and efficacy of a pozelimab and cemdisiran combination in adult patients with paroxysmal noctur…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
This document reports an update on a clinical trial testing iptacopan in Korean patients with Paroxysmal Nocturnal Hemoglobinuria or C3 glom…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
Clinical trial update analyzing long-term safety of Danicopan as add-on therapy in paroxysmal nocturnal hemoglobinuria using IPIG-Registry d…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria [Phase 1, Phase 2]
This entry reports on a Phase 1/2 clinical trial evaluating crovalimab in healthy volunteers and patients with paroxysmal nocturnal hemoglob…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
Clinical trial update on the long-term safety of danicopan in a registry-based cohort for paroxysmal nocturnal hemoglobinuria, currently act…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: National Longitudinal Cohort of Hematological Diseases
Recruiting for a national longitudinal cohort study of hematological diseases sponsored by the Institute of Hematology & Blood Diseases Hosp…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China [Phase 2]
Phase 2 clinical trial update for SLN12140 in adults with paroxysmal nocturnal hemoglobinuria in China; the study is not yet recruiting.
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CLINICAL_TRIALS research · 2026-01-28Clinical 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]
Phase 3 trial update evaluating safety, pharmacokinetics, and efficacy of crovalimab versus eculizumab in participants with paroxysmal noctu…
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Completed status for the Paroxysmal nocturnal hemoglobinuria (PNH) Registry, an observational study sponsored by Alexion Pharmaceuticals to …
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CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients. [Phase 2]
Phase 2 clinical trial update of LP-005 in paroxysmal nocturnal hemoglobinuria, reporting on efficacy, safety, and pharmacokinetics; the stu…
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CLINICAL_TRIALS research · 2025-12-22Clinical Trial Update: Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
A clinical trial update describing a secondary research study that analyzes molecular and clinical aspects of bone marrow failure and VEXAS …