Not applicableAvailableNCT05982938What this trial is testingDanicopan Early Access ProgramWho this might be right forParoxysmal Nocturnal HemoglobinuriaPNHExtravascular Hemolysis Alexion Pharmaceuticals, Inc.
Early research (Phase 1)Study completedNCT00012376What this trial is testingChemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid CancerWho this might be right forAccelerated Phase Chronic Myelogenous LeukemiaAdult Acute Myeloid Leukemia With 11q23 (MLL) AbnormalitiesAdult Acute Myeloid Leukemia With Del(5q)+15 more National Cancer Institute (NCI) 35
Testing effectiveness (Phase 2)Study completedNCT04965597What this trial is testingTreosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Who this might be right forBone Marrow Failure SyndromeCongenital Amegakaryocytic ThrombocytopeniaDiamond-Blackfan Anemia+6 more Fred Hutchinson Cancer Center 40
Not applicableStudy completedNCT01294891What this trial is testingMicrovascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell DiseaseWho this might be right forRheologic DiseaseSickle Cell DiseaseParoxysmal Nocturnal Hemoglobinuria Oregon Health and Science University 6
Testing effectiveness (Phase 2)Study completedNCT00004143What this trial is testingAllogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure SyndromesWho this might be right forSickle Cell AnemiaSevere Aplastic AnemiaParoxysmal Nocturnal Hemoglobinuria (PNH)+1 more David Rizzieri, MD 2
Large-scale testing (Phase 3)Study completedNCT03056040What this trial is testingALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabWho this might be right forParoxysmal Nocturnal Hemoglobinuria (PNH) Alexion Pharmaceuticals, Inc. 202
Early research (Phase 1)Study completedNCT05828485What this trial is testingEffect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult SubjectsWho this might be right forParoxysmal Nocturnal Hemoglobinuria Wuhan Createrna Science and Technology Co., Ltd 12
Large-scale testing (Phase 3)Study completedNCT03406507What this trial is testingRavulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal HemoglobinuriaWho this might be right forParoxysmal Nocturnal Hemoglobinuria Alexion Pharmaceuticals, Inc. 13
Large-scale testing (Phase 3)Study completedNCT02946463What this trial is testingALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)Who this might be right forParoxysmal Nocturnal Hemoglobinuria (PNH) Alexion Pharmaceuticals, Inc. 272
Testing effectiveness (Phase 2)Study completedNCT02605993What this trial is testingOpen-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)Who this might be right forParoxysmal Nocturnal HemoglobinuriaPNH Alexion Pharmaceuticals, Inc. 26
Testing effectiveness (Phase 2)Active Not RecruitingNCT00544115What this trial is testingDonor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other DisordersWho this might be right forChronic Myeloproliferative DisordersGraft Versus Host DiseaseLeukemia+5 more City of Hope Medical Center 260
Early research (Phase 1)Study completedNCT07035665What this trial is testingA Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy SubjectsWho this might be right forIgA NephropathyParoxysmal Nocturnal Hemoglobinuria Chengdu Suncadia Medicine Co., Ltd. 16
Not applicableStudy completedNCT05337683What this trial is testingA Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH PatientsWho this might be right forPNH Handok Inc. 80
Testing effectiveness (Phase 2)Study completedNCT03427060What this trial is testingCoversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 PolymorphismsWho this might be right forParoxysmal Nocturnal Hemoglobinuria (PNH) AKARI Therapeutics 1
Not applicableStudy completedNCT04671810What this trial is testingProspective Observational Study of Long-term Pathogenic Treatment of Elizaria®Who this might be right forParoxysmal Nocturnal Hemoglobinuria AO GENERIUM 44
Large-scale testing (Phase 3)Study completedNCT04558918What this trial is testingStudy of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentWho this might be right forParoxysmal Nocturnal Hemoglobinuria (PNH) Novartis Pharmaceuticals 97
Large-scale testing (Phase 3)Not Yet RecruitingNCT07816237What this trial is testingA 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 TherapyWho this might be right forParoxysmal Nocturnal Hemoglobinuria, PNH Bisirna Therapeutics (Suzhou) Co., Ltd. 70
Not applicableActive Not RecruitingNCT06903234What this trial is testingPost-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH RegistryWho this might be right forHemoglobinuria, Paroxysmal Novartis Pharmaceuticals 200
Large-scale testing (Phase 3)Study completedNCT04463056What this trial is testingEfficacy and Safety of Elizaria® vs. Soliris® in Patients With PNHWho this might be right forParoxysmal Nocturnal HemoglobinuriaMarchiafava-Micheli SyndromeParoxysmal Hemoglobinuria AO GENERIUM 32
Post-approval studies (Phase 4)Study completedNCT04320602What this trial is testingRavulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose EculizumabWho this might be right forParoxysmal Nocturnal Hemoglobinuria Alexion Pharmaceuticals, Inc. 18