| Recruiting | A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants NCT07462780 | Shenzhen Kangzhe Biotechnology Co., Ltd. | Phase 1 |
| Recruiting | A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Fa NCT07187401 | Regeneron Pharmaceuticals | Phase 1 / Phase 2 |
| Not Yet Recruiting | SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China NCT07387302 | Linno Pharmaceuticals, Inc. | Phase 2 |
| Recruiting | REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria NCT07229235 | Novartis Pharmaceuticals | — |
| Not Yet Recruiting | Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT06134414 | Wuhan Createrna Science and Technology Co., Ltd | Phase 2 |
| Recruiting | Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients NCT06934967 | Novartis Pharmaceuticals | Phase 3 |
| Not Yet Recruiting | Hematological Disorders in EHPVO Patients NCT07108023 | Rahab Nady | — |
| Recruiting | Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment NCT07152288 | Haisco Pharmaceutical Group Co., Ltd. | Phase 1 |
| Not Yet Recruiting | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of o NCT06848296 | Visirna Therapeutics HK Limited | — |
| Recruiting | Study of Ultomiris® (Ravulizumab) Safety in Pregnancy NCT06312644 | Alexion Pharmaceuticals, Inc. | — |
| Recruiting | Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT06933914 | Wuhan Createrna Science and Technology Co., Ltd | Phase 2 / Phase 3 |
| Completed | Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects NCT06852092 | Haisco Pharmaceutical Group Co., Ltd. | Phase 1 |
| Completed | A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（P NCT06561841 | Haisco Pharmaceutical Group Co., Ltd. | Phase 2 |
| Completed | A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants NCT06294301 | Longbio Pharma | Phase 1 |
| Active Not Recruiting | Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PN NCT05876312 | ADARx Pharmaceuticals, Inc. | Phase 1 / Phase 2 |
| Terminated | BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Respons NCT05116774 | BioCryst Pharmaceuticals | Phase 2 |
| Terminated | BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Com NCT05116787 | BioCryst Pharmaceuticals | Phase 2 |
| Completed | Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Com NCT04820530 | Novartis Pharmaceuticals | Phase 3 |
| Recruiting | A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT04901936 | Apellis Pharmaceuticals, Inc. | Phase 2 |
| Completed | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Ant NCT04558918 | Novartis Pharmaceuticals | Phase 3 |
| Recruiting | National Longitudinal Cohort of Hematological Diseases NCT04645199 | Institute of Hematology & Blood Diseases Hospital, China | — |
| Completed | An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical S NCT06987864 | Biocad | Phase 3 |
| Terminated | Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy NCT04170023 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PN NCT03946748 | Regeneron Pharmaceuticals | Phase 2 |
| Recruiting | Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood St NCT03520647 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 NCT03588026 | AKARI Therapeutics | Phase 3 |
| Completed | Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms NCT03427060 | AKARI Therapeutics | Phase 2 |
| Completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculiz NCT03472885 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Terminated | Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study NCT03225287 | Ra Pharmaceuticals, Inc. | Phase 2 |
| Completed | ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Trea NCT03056040 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Completed | Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Respon NCT03030183 | Ra Pharmaceuticals | Phase 2 |
| Completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT03053102 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients NCT03078582 | Ra Pharmaceuticals | Phase 2 |
| Completed | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Parox NCT02946463 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Completed | A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects Wit NCT02264639 | Apellis Pharmaceuticals, Inc. | Phase 1 |
| Completed | A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH NCT02352493 | Alnylam Pharmaceuticals | Phase 1 / Phase 2 |
| Terminated | Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT01335165 | Alexion Pharmaceuticals, Inc. | Phase 1 |
| Completed | Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies NCT00145613 | St. Jude Children's Research Hospital | Phase 2 |
| Completed | Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablat NCT00587054 | Memorial Sloan Kettering Cancer Center | Phase 2 |
| Completed | Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syn NCT00004143 | David Rizzieri, MD | Phase 2 |
| Available | Managed Access Programs for LNP023, Iptacopan NCT05222412 | Novartis Pharmaceuticals | — |