| Not Yet Recruiting | Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris NCT05646563 | NovelMed Therapeutics | Phase 2 |
| Not Yet Recruiting | A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy NCT07416162 | Novartis Pharmaceuticals | — |
| Not Yet Recruiting | A Long-term Efficacy and Safety of NTQ5082 Capsules NCT07177872 | Nanjing Chia-tai Tianqing Pharmaceutical | Phase 3 |
| Recruiting | Danicopan PMS in Korea NCT07457151 | AstraZeneca | — |
| Not Yet Recruiting | Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy NCT05646524 | NovelMed Therapeutics | Phase 2 |
| Recruiting | A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Noc NCT07154745 | Regeneron Pharmaceuticals | Phase 3 |
| Recruiting | Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT07470762 | Jiangsu Hansoh Pharmaceutical Co., Ltd. | Phase 1 / Phase 2 |
| Active Not Recruiting | Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: NCT07413250 | Alexion Pharmaceuticals, Inc. | — |
| Not Yet Recruiting | Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuri NCT07266155 | Longbio Pharma | Phase 2 |
| Not Yet Recruiting | A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients NCT07177859 | Nanjing Chia-tai Tianqing Pharmaceutical | Phase 3 |
| Active Not Recruiting | Long-term Safety of Danicopan: IPIG Registry-based Cohort Study NCT07413679 | Alexion Pharmaceuticals, Inc. | — |
| Recruiting | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Hav NCT06449001 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Terminated | NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice NCT07036718 | Novartis Pharmaceuticals | — |
| Completed | A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Health NCT07035665 | Chengdu Suncadia Medicine Co., Ltd. | Phase 1 |
| Recruiting | A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in C NCT06931691 | Novartis Pharmaceuticals | — |
| Active Not Recruiting | A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients NCT06764303 | Nanjing Chia-tai Tianqing Pharmaceutical | Phase 2 |
| Completed | Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment NCT06715943 | Chengdu Suncadia Medicine Co., Ltd. | Phase 3 |
| Recruiting | Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT06745622 | Haisco Pharmaceutical Group Co., Ltd. | Phase 2 |
| Completed | A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH NCT06593938 | Chengdu Suncadia Medicine Co., Ltd. | Phase 3 |
| Completed | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Part NCT06578949 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Recruiting | Specified Drug-use Surveillance of Fabhalta Capsules NCT06606314 | Novartis Pharmaceuticals | — |
| Recruiting | Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment NCT06932471 | Wuhan Createrna Science and Technology Co., Ltd | Phase 3 |
| Not Yet Recruiting | To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects NCT06543459 | Wuhan Createrna Science and Technology Co., Ltd | Phase 1 |
| Recruiting | Home Reported Outcomes in PNH NCT06411626 | Novartis Pharmaceuticals | — |
| Recruiting | MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophospham NCT06412497 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Recruiting | The International PNH Interest Group PNH Registry NCT06524726 | International PNH Interest Group | — |
| Recruiting | Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria NCT06298955 | Omeros Corporation | Phase 2 |
| Active Not Recruiting | Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT06238544 | Chengdu Suncadia Medicine Co., Ltd. | Phase 2 |
| Completed | Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemogl NCT06051357 | Chengdu Suncadia Medicine Co., Ltd. | Phase 2 |
| Completed | A Real-world, Multi-center, Prospective, Observational Study for PNH in China NCT06154512 | AstraZeneca | — |
| Completed | Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT06100900 | BioCryst Pharmaceuticals | Phase 1 |
| Completed | A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT06050226 | Wuhan Createrna Science and Technology Co., Ltd | Phase 2 |
| Completed | Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China NCT05886244 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Recruiting | A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT05776472 | Swedish Orphan Biovitrum | — |
| Completed | Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 W NCT05630001 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to R NCT05972967 | Omeros Corporation | Phase 2 |
| Recruiting | A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatm NCT05744921 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an NCT05842486 | Assistance Publique - Hôpitaux de Paris | — |
| Terminated | Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria NCT05741346 | BioCryst Pharmaceuticals | Phase 2 |
| Completed | A Study of Multiple Ascending Doses MY008211A in Healthy Adults NCT05828472 | Wuhan Createrna Science and Technology Co., Ltd | Phase 1 |
| Unknown | Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT05889299 | Omeros Corporation | Phase 1 |
| Active Not Recruiting | To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 NCT05476887 | Kira Pharmacenticals (US), LLC. | Phase 2 |
| Active Not Recruiting | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C NCT05389449 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Terminated | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab NCT05131204 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects NCT05828485 | Wuhan Createrna Science and Technology Co., Ltd | Phase 1 |
| Recruiting | A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Pati NCT05133531 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria NCT05274633 | Alexion Pharmaceuticals, Inc. | — |
| Completed | A Study of Single-dose MY008211A in Healthy Adults NCT05642585 | Wuhan Createrna Science and Technology Co., Ltd | Phase 1 |
| Completed | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marro NCT04965597 | Fred Hutchinson Cancer Center | Phase 2 |
| Completed | A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults NCT06326814 | Sanofi | Phase 1 |
| Completed | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria NCT04811716 | Regeneron Pharmaceuticals | Phase 2 |
| Active Not Recruiting | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT04747613 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Wh NCT04888507 | Regeneron Pharmaceuticals | Phase 2 |
| Recruiting | Global PNH Patient Registry NCT05755867 | Aplastic Anemia and MDS International Foundation | — |
| Completed | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Ec NCT04320602 | Alexion Pharmaceuticals, Inc. | Phase 4 |
| Active Not Recruiting | A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants W NCT04654468 | Hoffmann-La Roche | Phase 3 |
| Completed | The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT04079257 | Radboud University Medical Center | — |
| Completed | To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple NCT05490017 | Kira Pharmacenticals (US), LLC. | Phase 1 |
| Terminated | A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT04702568 | BioCryst Pharmaceuticals | Phase 2 |
| Completed | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Ha NCT04469465 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Active Not Recruiting | A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Noc NCT04434092 | Hoffmann-La Roche | Phase 3 |
| Active Not Recruiting | A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants NCT04432584 | Hoffmann-La Roche | Phase 3 |
| Completed | First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH NCT04330534 | BioCryst Pharmaceuticals | Phase 1 / Phase 2 |
| Terminated | REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy NCT04162470 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria NCT04128943 | University Hospital, Basel, Switzerland | — |
| Completed | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH NCT04085601 | Apellis Pharmaceuticals, Inc. | Phase 3 |
| Completed | A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haem NCT04058158 | Samsung Bioepis Co., Ltd. | Phase 3 |
| Completed | Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® NCT04671810 | AO GENERIUM | — |
| Completed | Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patien NCT03896152 | Novartis Pharmaceuticals | Phase 2 |
| Completed | Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria NCT04060264 | Biocad | Phase 3 |
| Unknown | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH NCT03866681 | Peking Union Medical College Hospital | Phase 4 |
| Completed | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglob NCT03748823 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Completed | A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH NCT03818607 | Amgen | Phase 3 |
| Unknown | Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS NCT03329365 | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | — |
| Completed | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT03500549 | Apellis Pharmaceuticals, Inc. | Phase 3 |
| Completed | A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) NCT04679103 | AO GENERIUM | Phase 3 |
| Completed | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria NCT03406507 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Terminated | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Pa NCT03333486 | Roswell Park Cancer Institute | Phase 2 |
| Completed | Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH NCT04463056 | AO GENERIUM | Phase 3 |
| Completed | A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT03181633 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Terminated | rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study NCT03829449 | AKARI Therapeutics | Phase 3 |
| Active Not Recruiting | Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF NCT03047746 | Children's Hospital of Philadelphia | N/A |
| Completed | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal NCT02605993 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH S NCT02588833 | Apellis Pharmaceuticals, Inc. | Phase 1 |
| Completed | Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysm NCT02534909 | Novartis Pharmaceuticals | Phase 2 |
| Terminated | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies NCT02179359 | Masonic Cancer Center, University of Minnesota | N/A |
| Unknown | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturn NCT01760096 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Unknown | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal H NCT01642979 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Completed | Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study NCT01412047 | Alexion Pharmaceuticals, Inc. | — |
| Completed | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem NCT01529827 | Roswell Park Cancer Institute | Phase 2 |
| Completed | Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease NCT01294891 | Oregon Health and Science University | — |
| Completed | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With NCT00856388 | Roswell Park Cancer Institute | N/A |
| Completed | Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Trea NCT00731328 | Asan Medical Center | Phase 2 |
| Completed | E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients NCT01194804 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients NCT01192399 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treati NCT00397813 | Fred Hutchinson Cancer Center | Phase 2 |
| Completed | Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies NCT00143559 | St. Jude Children's Research Hospital | Phase 2 |
| Completed | Unrelated Donor Stem Cell Transplantation NCT01364363 | Scripps Health | N/A |
| Completed | Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry NCT01374360 | Alexion Pharmaceuticals, Inc. | — |
| Completed | Nonmyeloablative Allogeneic Transplant NCT01272817 | Scripps Health | N/A |
| Completed | Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer NCT00012376 | National Cancer Institute (NCI) | Phase 1 |
| No Longer Available | Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemogl NCT06028594 | Regeneron Pharmaceuticals | — |
| Available | Danicopan Early Access Program NCT05982938 | Alexion Pharmaceuticals, Inc. | — |