Records containing: Paroxysmal Nocturnal Hemoglobinuria

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• CLINICAL_TRIALS study record update · 2025-02-20
  Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
• CLINICAL_TRIALS study record update · 2025-01-16
  A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
• CLINICAL_TRIALS study record update · 2025-01-08
  An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects with Paroxysmal Nocturnal Hemoglobinuria
• CLINICAL_TRIALS study record update · 2024-12-20
  A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
• CLINICAL_TRIALS study record update · 2024-12-13
  A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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• CLINICAL_TRIALS study record update · 2024-11-01
  An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• CLINICAL_TRIALS study record update · 2024-10-28
  An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Complement Inhibitor-naïve Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• CLINICAL_TRIALS study record update · 2024-10-15
  An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• CLINICAL_TRIALS study record update · 2024-10-08
  Paroxysmal Nocturnal Hemoglobinuria and Embolic Strokes of Undetermined Source (ESUS) and Transient Ischemic Attacks of Undetermined Source (ETUS)
• CLINICAL_TRIALS study record update · 2024-09-19
  A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Currently Treated With Eculizumab or Ravulizumab
• CLINICAL_TRIALS study record update · 2024-08-12
  Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
• CLINICAL_TRIALS study record update · 2024-08-09
  A Single-center, Randomized, Double-blind, Single Oral Dose, Placebo-controlled Study to Evaluate the Effect of MY008211A Tablets on QTc Interval in Healthy Chinese Adult Subjects
• CLINICAL_TRIALS study record update · 2024-07-29
  The International PNH Interest Group PNH Registry
• CLINICAL_TRIALS study record update · 2024-04-02
  A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells
• CLINICAL_TRIALS study record update · 2024-03-26
  A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria
• CLINICAL_TRIALS study record update · 2024-03-22
  A First-in-human, Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Ascending Single Doses of SAR443809 in Healthy Adult Subjects
• CLINICAL_TRIALS study record update · 2024-03-07
  An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• CLINICAL_TRIALS study record update · 2023-12-18
  A Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis
• CLINICAL_TRIALS study record update · 2023-09-13
  Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Paroxysmal Nocturnal Hemoglobinuria.
• CLINICAL_TRIALS study record update · 2023-09-08
  A Phase I, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Doses Clinical Study to Evaluate the Safety, Tolerability and Pharmacokinetic Characteristics of MY008211A Tablets in Healthy Adult Volunteers
• CLINICAL_TRIALS study record update · 2023-09-08
  A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Doses Clinical Study to Evaluate the Safety, Tolerability and Pharmacokinetic/Pharmacodynamics Characteristics of MY008211A Tablets in Healthy Adult Volunteers
• CLINICAL_TRIALS study record update · 2023-07-24
  A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
• CLINICAL_TRIALS study record update · 2023-06-12
  An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
• CLINICAL_TRIALS study record update · 2023-06-05
  A Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
• CLINICAL_TRIALS study record update · 2023-06-05
  A Phase 1b Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria