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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
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 WorksHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingPhase 32026-09-28NCT05744921
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaEnrolling by invitationNot applicable2026-09-25NCT07657052
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUSCytopenia, Embolism and Thrombosis, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Intracranial Embolism and Thrombosis, Thromboembolism, anemia, bone marrow disorder, brain disorder, brain ischemia, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hematologic disorder, hemolytic anemia, intracranial thrombosis, myelodysplastic syndrome, nervous system disorder, paroxysmal nocturnal hemoglobinuria, transient ischemic attack, vascular disorderActive, not recruitingNot stated2026-09-18NCT03329365
Specified Drug-use Surveillance of Fabhalta CapsulesHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot stated2026-09-10NCT06606314
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot stated2026-09-03NCT05776472
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-08-26NCT04654468
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitorsHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-08-18NCT04434092
Danicopan PMS in KoreaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingNot stated2026-08-14NCT07457151
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-08-07NCT04747613
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaNot yet recruitingPhase 32026-08-05NCT07747090
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingPhase 32026-08-05NCT07154745
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement InhibitorsHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-07-28NCT04432584
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 22026-07-16NCT06298955
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 GlomerulopathyFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Hemolysis, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, anemia, bone marrow disorder, complement 3 glomerulopathy, glomerulonephritis, hematologic disorder, hemolytic anemia, immune system disorder, kidney disorder, membranoproliferative glomerulonephritis, myelodysplastic syndrome, nephritis, paroxysmal nocturnal hemoglobinuria, proteinuria, urinary system disorderRecruitingNot stated2026-06-26NCT07416162
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNHHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Hemolysis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-06-18NCT05389449
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMFBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Infant, Newborn, Diseases, acquired aplastic anemia, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot applicable2026-06-17NCT03047746
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.Acquired amegakaryocytic thrombocytopenia, Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingPhase 22026-06-03NCT06412497
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in ChinaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingNot stated2026-06-01NCT06931691
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 TreatmentHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-04-30NCT05133531
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingPhase 1 / Phase 22026-04-13NCT07470762
Study of NM8074 in Adult PNH Patients With Inadequate Response to SolirisHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaNot yet recruitingPhase 22026-04-13NCT05646563
Long-term Safety of Danicopan: IPIG Registry-based Cohort StudyHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot stated2026-02-17NCT07413679
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry DataHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot stated2026-02-17NCT07413250
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 22025-12-24NCT06238544
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular HemolysisExtravascular Hemolysis, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Hemolysis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaRecruitingPhase 32025-12-23NCT06449001