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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
Real-life Treatment Outcomes of Ravulizumab in PNHHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeActive, not recruitingNot stated2026-09-23NCT06633536
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, pure red-cell aplasiaRecruitingPhase 22026-09-22NCT01174108
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
A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor TherapyHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeNot yet recruitingPhase 32026-09-11NCT07816237
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
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 22026-09-08NCT03520647
Study of SGB-9768 in Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeNot yet recruitingPhase 22026-09-04NCT07796256
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
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 12026-09-01NCT07760467
Efficacy and Safety of Lanoracopan in the Treatment of PNHHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeNot yet recruitingNot stated2026-08-26NCT07788105
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
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH PatientsHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 32026-08-07NCT06934967
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 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 InhibitorHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 1 / Phase 22026-07-09NCT07187401
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 Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 22026-06-26NCT04901936
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal HemoglobinuriaHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingNot stated2026-06-25NCT07229235