Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS research · 2025-12-05Clinical Trial Update: Managed Access Programs for LNP023, Iptacopan
This clinical trial update notes the availability of managed access programs for iptacopan (LNP023) for patients with C3 glomerulopathy and …
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CLINICAL_TRIALS research · 2025-12-05Clinical Trial Update: Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial evaluating the efficacy, safety, and pharmacokinetics of LP-005 injection in individuals with paroxysmal nocturnal …
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
A multi-center ambispective cohort study in China evaluating the impact of iptacopan for adults with paroxysmal nocturnal hemoglobinuria. Th…
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1]
A Phase Ib, multicenter, open-label study to evaluate multiple-dose EA5 in adults with paroxysmal nocturnal hemoglobinuria is currently recr…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 2]
This is a Phase 2 study evaluating NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria. The study is currently active but not recruitin…
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CLINICAL_TRIALS research · 2025-09-17Clinical Trial Update: A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 3]
A Phase III study of NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria is planned, with the trial currently not yet recruiting.
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CLINICAL_TRIALS research · 2025-09-17Clinical Trial Update: A Long-term Efficacy and Safety of NTQ5082 Capsules [Phase 3]
This update describes a Phase 3 long-term efficacy and safety study of NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria, currently n…
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CLINICAL_TRIALS research · 2025-09-03Clinical Trial Update: Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment [Phase 1]
Phase 1 study evaluating the pharmacokinetics, safety, and pharmacodynamics of the investigational drug HSK39297 in subjects with hepatic im…
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CLINICAL_TRIALS research · 2025-08-27Clinical Trial Update: Home Reported Outcomes in PNH
Clinical trial update indicating a Novartis-sponsored study on home-reported outcomes in paroxysmal nocturnal hemoglobinuria is currently re…
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CLINICAL_TRIALS research · 2025-08-06Clinical Trial Update: Hematological Disorders in EHPVO Patients
A clinical trial update describes a study on hematological disorders in patients with Extrahepatic portal vein obstruction and thrombophilia…
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CLINICAL_TRIALS research · 2025-07-29Clinical Trial Update: Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial investigating the safety and efficacy of MY008211A in patients with paroxysmal nocturnal hemoglobinuria (PNH) is li…
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CLINICAL_TRIALS research · 2025-07-16Clinical Trial Update: Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF [NA]
Clinical trial update on unrelated and partially matched related donor PSCT with TCR αβ depletion for bone marrow failure conditions; the st…
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CLINICAL_TRIALS research · 2025-06-19Clinical Trial Update: MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. [Phase 2]
Phase 2 trial evaluating hematopoietic cell transplantation with reduced-intensity conditioning and post-transplant cyclophosphamide for sev…
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CLINICAL_TRIALS research · 2025-06-11Clinical Trial Update: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial update indicates that pegcetacoplan for pediatric patients with paroxysmal nocturnal hemoglobinuria is currently re…
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CLINICAL_TRIALS research · 2025-05-18Clinical Trial Update: A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase II clinical trial evaluating the efficacy and safety of XH-S003 capsules in patients with paroxysmal nocturnal hemoglobinuria, current…
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CLINICAL_TRIALS research · 2025-05-14Clinical Trial Update: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This clinical trial update notes that a real-world effectiveness study of pegcetacoplan for Paroxysmal nocturnal hemoglobinuria is currently…
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CLINICAL_TRIALS research · 2025-05-09Clinical Trial Update: Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment [Phase 3]
Phase 3 clinical trial evaluating the safety and efficacy of MY008211A in patients with Paroxysmal nocturnal hemoglobinuria who have residua…
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CLINICAL_TRIALS research · 2025-04-18Clinical Trial Update: Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2, Phase 3]
A long-term safety and tolerability study of MY008211A tablets in paroxysmal nocturnal hemoglobinuria is enrolling in Phase 2 and Phase 3. T…
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CLINICAL_TRIALS research · 2025-03-30Clinical Trial Update: Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 study evaluating NM8074 in patients with paroxysmal nocturnal hemoglobinuria who are already treated with Soliris; the trial is not …
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CLINICAL_TRIALS research · 2025-02-27Clinical Trial Update: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of of VSA012 in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH)
This is an early-phase clinical trial update evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary effica…
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CLINICAL_TRIALS research · 2025-02-20Clinical Trial Update: Global PNH Patient Registry
Global patient registry for Paroxysmal Nocturnal Hemoglobinuria is currently recruiting. The registry aims to collect natural history and ou…
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CLINICAL_TRIALS research · 2024-12-20Clinical Trial Update: Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 clinical trial assessing long-term safety and tolerability of HSK39297 in patients with paroxysmal nocturnal hemoglobinuria is cur…
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CLINICAL_TRIALS research · 2024-10-28Clinical Trial Update: To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 [Phase 2]
A Phase 2 clinical trial is evaluating KP104 for safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in Paroxysmal noctur…
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CLINICAL_TRIALS research · 2024-10-08Clinical Trial Update: Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
An update on a clinical trial exploring paroxysmal nocturnal hemoglobinuria in embolic strokes of undetermined source (ESUS/ETUS) and relate…
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CLINICAL_TRIALS research · 2024-07-29Clinical Trial Update: The International PNH Interest Group PNH Registry
This clinical trial update describes an ongoing PNH patient registry led by the International PNH Interest Group to collect data on Paroxysm…