| BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy (NCT05116787) | BioCryst Pharmaceuticals | 2021-10-26 |
| Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (NCT04820530) | Novartis Pharmaceuticals | 2021-07-19 |
| A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT04901936) | Apellis Pharmaceuticals, Inc. | 2021-02-04 |
| Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (NCT04558918) | Novartis Pharmaceuticals | 2021-01-25 |
| National Longitudinal Cohort of Hematological Diseases (NCT04645199) | Institute of Hematology & Blood Diseases Hospital, China | 2020-12-01 |
| An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN (NCT06987864) | Biocad | 2020-04-13 |
| Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy (NCT04170023) | Alexion Pharmaceuticals, Inc. | 2019-12-16 |
| Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT03946748) | Regeneron Pharmaceuticals | 2019-05-16 |
| Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis (NCT03520647) | National Heart, Lung, and Blood Institute (NHLBI) | 2019-02-19 |
| Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 (NCT03588026) | AKARI Therapeutics | 2018-06-07 |
| Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms (NCT03427060) | AKARI Therapeutics | 2018-05-14 |
| Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab (NCT03472885) | Alexion Pharmaceuticals, Inc. | 2018-05-08 |
| Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study (NCT03225287) | Ra Pharmaceuticals, Inc. | 2017-07-17 |
| ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab (NCT03056040) | Alexion Pharmaceuticals, Inc. | 2017-05-17 |
| Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT03053102) | Alexion Pharmaceuticals, Inc. | 2017-03-31 |
| Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients (NCT03078582) | Ra Pharmaceuticals | 2017-03-08 |
| ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT02946463) | Alexion Pharmaceuticals, Inc. | 2016-12-12 |
| A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH (NCT02264639) | Apellis Pharmaceuticals, Inc. | 2015-02-23 |
| Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT01335165) | Alexion Pharmaceuticals, Inc. | 2011-04-01 |
| Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies (NCT00145613) | St. Jude Children's Research Hospital | 2003-06-01 |
| Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders (NCT00587054) | Memorial Sloan Kettering Cancer Center | 2001-06-01 |
| Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes (NCT00004143) | David Rizzieri, MD | 1999-09-01 |
| Managed Access Programs for LNP023, Iptacopan (NCT05222412) | Novartis Pharmaceuticals | |