AstraZeneca: Hemoglobinuria, Paroxysmal trials

The registry lists 35 Hemoglobinuria, Paroxysmal studies with AstraZeneca as lead sponsor. 4 are open to participants now and 18 have results posted.

Phase 1 2Phase 2 8Phase 3 12Phase 4 2No phase 12(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Danicopan PMS in KoreaRecruitingNCT07457151
• Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular HemolysisRecruitingNCT06449001Phase 3
• Study of Ultomiris® (Ravulizumab) Safety in PregnancyRecruitingNCT06312644
• Danicopan Early Access ProgramAvailableNCT05982938
• Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT06578949Phase 3Results posted
• Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in ChinaCompletedNCT05886244Phase 3Results posted
• Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)CompletedNCT04469465Phase 3Results posted
• Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose EculizumabCompletedNCT04320602Phase 4Results posted
• Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as MonotherapyTerminatedNCT04170023Phase 2Results posted
• Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCompletedNCT03748823Phase 3Results posted
• Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to EculizumabCompletedNCT03472885Phase 2Results posted
• A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT03406507Phase 3Results posted
• A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03181633Phase 2Results posted
• ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCompletedNCT03056040Phase 3Results posted
• Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03053102Phase 2Results posted
• ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT02946463Phase 3Results posted
• Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT02605993Phase 2Results posted
• Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT02598583Phase 1/2Results posted
• E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsCompletedNCT01194804Phase 2Results posted
• Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria PatientsCompletedNCT01192399Phase 2Results posted
• Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT00867932Phase 4Results posted
• Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT00122317Phase 3Results posted
• Long-term Safety of Danicopan: IPIG Registry-based Cohort StudyActive, not recruitingNCT07413679
• Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry DataActive, not recruitingNCT07413250
• Real-life Treatment Outcomes of Ravulizumab in PNHActive, not recruitingNCT06633536
• A Real-world, Multi-center, Prospective, Observational Study for PNH in ChinaCompletedNCT06154512
• A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNHActive, not recruitingNCT05389449Phase 3
• Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT05274633
• Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies StudyCompletedNCT01412047
• Paroxysmal Nocturnal Hemoglobinuria (PNH) RegistryCompletedNCT01374360
• Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)TerminatedNCT01335165Phase 1
• Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure SyndromesCompletedNCT01192425
• The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment ProtocolApproved for marketingNCT00438789
• Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsCompletedNCT00122330Phase 3
• Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With EculizumabCompletedNCT00122304Phase 3