Hemoglobinuria, Paroxysmal: all 192 studies

Page 1 of 2. Open to participants first (49), then studies with results posted, then the rest; newest registration first within each.

• Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic DataRecruitingNCT07877727
• 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 TherapyNot yet recruitingNCT07816237Phase 3
• Study of SGB-9768 in Patients With Paroxysmal Nocturnal HemoglobinuriaNot yet recruitingNCT07796256Phase 2
• Efficacy and Safety of Lanoracopan in the Treatment of PNHNot yet recruitingNCT07788105
• To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.RecruitingNCT07760467Phase 1
• A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.Not yet recruitingNCT07747090Phase 3
• A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal HemoglobinuriaEnrolling by invitationNCT07657052Not applicable
• Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT07470762Phase 1/2
• A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy ParticipantsRecruitingNCT07462780Phase 1
• Danicopan PMS in KoreaRecruitingNCT07457151
• A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 GlomerulopathyRecruitingNCT07416162
• SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in ChinaNot yet recruitingNCT07387302Phase 2
• Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT07266155Phase 2
• A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT07256301Phase 1
• REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT07229235
• 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 InhibitorRecruitingNCT07187401Phase 1/2
• A Long-term Efficacy and Safety of NTQ5082 CapsulesNot yet recruitingNCT07177872Phase 3
• A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria PatientsNot yet recruitingNCT07177859Phase 3
• A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyRecruitingNCT07154745Phase 3
• Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic ImpairmentRecruitingNCT07152288Phase 1
• Hematological Disorders in EHPVO PatientsNot yet recruitingNCT07108023
• Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research StudyEnrolling by invitationNCT07102849
• A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT06978699Phase 2
• Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH PatientsRecruitingNCT06934967Phase 3
• Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT06933914Phase 2/3
• Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor TherapyRecruitingNCT06932744Phase 3
• Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentRecruitingNCT06932471Phase 3
• A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in ChinaRecruitingNCT06931691
• Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingNCT06745622Phase 2
• To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy SubjectsNot yet recruitingNCT06543459Phase 1
• The International PNH Interest Group PNH RegistryRecruitingNCT06524726
• Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular HemolysisRecruitingNCT06449001Phase 3
• 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.RecruitingNCT06412497Phase 2
• Home Reported Outcomes in PNHRecruitingNCT06411626
• Study of Ultomiris® (Ravulizumab) Safety in PregnancyRecruitingNCT06312644
• Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT06134414Phase 2
• Danicopan Early Access ProgramAvailableNCT05982938
• Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsRecruitingNCT05876312Phase 1/2
• Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure DisordersRecruitingNCT05794425Phase 1/2
• Global PNH Patient RegistryRecruitingNCT05755867
• 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 WorksRecruitingNCT05744921Phase 3
• Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Not yet recruitingNCT05731050Phase 2
• Study of NM8074 in Adult PNH Patients With Inadequate Response to SolirisNot yet recruitingNCT05646563Phase 2
• Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyNot yet recruitingNCT05646524Phase 2
• Managed Access Programs for LNP023, IptacopanAvailableNCT05222412
• A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingNCT04901936Phase 2
• National Longitudinal Cohort of Hematological DiseasesRecruitingNCT04645199
• Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisRecruitingNCT03520647Phase 2
• 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-cellsRecruitingNCT01174108Phase 2
• 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
• Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal HemoglobinuriaTerminatedNCT05741346Phase 2Results posted
• Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.CompletedNCT05630001Phase 3Results posted
• Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaTerminatedNCT05131204Phase 3Results posted
• BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor TherapyTerminatedNCT05116787Phase 2Results posted
• BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor TherapyTerminatedNCT05116774Phase 2Results posted
• Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab TherapyCompletedNCT04888507Phase 2Results posted
• Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyCompletedNCT04820530Phase 3Results posted
• Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab MonotherapyCompletedNCT04811716Phase 2Results posted
• A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)TerminatedNCT04702568Phase 2Results posted
• A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionActive, not recruitingNCT04654468Phase 3Results posted
• Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentCompletedNCT04558918Phase 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
• A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitorsActive, not recruitingNCT04434092Phase 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
• REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.TerminatedNCT04162470Phase 3Results posted
• A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNHCompletedNCT04085601Phase 3Results posted
• A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal HaemoglobinuriaCompletedNCT04058158Phase 3Results posted
• Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03946748Phase 2Results posted
• Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT03896152Phase 2Results posted
• rVA576 (Coversin) Long Term Safety and Efficacy Surveillance StudyTerminatedNCT03829449Phase 3Results posted
• A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNHCompletedNCT03818607Phase 3Results posted
• Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCompletedNCT03748823Phase 3Results posted
• A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNHCompletedNCT03593200Phase 2Results posted
• Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03500549Phase 3Results posted
• Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to EculizumabCompletedNCT03472885Phase 2Results posted
• Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03439839Phase 2Results posted
• Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsCompletedNCT03427060Phase 2Results posted
• A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT03406507Phase 3Results posted
• Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerTerminatedNCT03333486Phase 2Results posted
• Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical StudyTerminatedNCT03225287Phase 2Results posted
• A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03181633Phase 2Results posted
• Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH PatientsCompletedNCT03078582Phase 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
• Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to EculizumabCompletedNCT03030183Phase 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
• Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)CompletedNCT02591862Phase 2Results posted
• Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.CompletedNCT02588833Phase 1Results posted
• Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal HemoglobinuriaCompletedNCT02534909Phase 2Results posted
• A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHCompletedNCT02352493Phase 1/2Results posted
• A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNHCompletedNCT02264639Phase 1Results posted
• Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic AnemiaCompletedNCT02224872Phase 2Results posted
• Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesCompletedNCT01529827Phase 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
• Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure StatesCompletedNCT00997386Phase 2Results posted

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