| Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT05274633) | Alexion Pharmaceuticals, Inc. | 2022-06-17 |
| A Study of Single-dose MY008211A in Healthy Adults (NCT05642585) | Wuhan Createrna Science and Technology Co., Ltd | 2022-05-27 |
| Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) (NCT04965597) | Fred Hutchinson Cancer Center | 2022-04-19 |
| A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults (NCT06326814) | Sanofi | 2021-10-11 |
| Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy (NCT04811716) | Regeneron Pharmaceuticals | 2021-07-29 |
| Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT04747613) | Novartis Pharmaceuticals | 2021-07-27 |
| Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy (NCT04888507) | Regeneron Pharmaceuticals | 2021-07-08 |
| Global PNH Patient Registry (NCT05755867) | Aplastic Anemia and MDS International Foundation | 2021-05-06 |
| Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab (NCT04320602) | Alexion Pharmaceuticals, Inc. | 2021-04-14 |
| A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition (NCT04654468) | Hoffmann-La Roche | 2021-03-17 |
| The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT04079257) | Radboud University Medical Center | 2021-02-23 |
| To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 (NCT05490017) | Kira Pharmacenticals (US), LLC. | 2020-12-30 |
| A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT04702568) | BioCryst Pharmaceuticals | 2020-12-18 |
| Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) (NCT04469465) | Alexion Pharmaceuticals, Inc. | 2020-12-16 |
| A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors (NCT04434092) | Hoffmann-La Roche | 2020-10-08 |
| First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH (NCT04330534) | BioCryst Pharmaceuticals | 2020-03-03 |
| REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. (NCT04162470) | Regeneron Pharmaceuticals | 2019-12-03 |
| Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (NCT04128943) | University Hospital, Basel, Switzerland | 2019-11-01 |
| A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH (NCT04085601) | Apellis Pharmaceuticals, Inc. | 2019-08-27 |
| A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria (NCT04058158) | Samsung Bioepis Co., Ltd. | 2019-08-07 |
| Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® (NCT04671810) | AO GENERIUM | 2019-06-13 |
| Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT03896152) | Novartis Pharmaceuticals | 2019-04-05 |
| Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT04060264) | Biocad | 2019-04-04 |
| Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH (NCT03866681) | Peking Union Medical College Hospital | 2019-04-01 |
| Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab (NCT03748823) | Alexion Pharmaceuticals, Inc. | 2019-02-19 |
| A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (NCT03818607) | Amgen | 2019-01-22 |
| Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS (NCT03329365) | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | 2018-11-01 |
| A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) (NCT04679103) | AO GENERIUM | 2018-05-03 |
| A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (NCT03406507) | Alexion Pharmaceuticals, Inc. | 2018-02-22 |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer (NCT03333486) | Roswell Park Cancer Institute | 2017-12-07 |
| Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH (NCT04463056) | AO GENERIUM | 2017-11-29 |
| A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT03181633) | Alexion Pharmaceuticals, Inc. | 2017-06-22 |
| rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study (NCT03829449) | AKARI Therapeutics | 2017-03-13 |
| Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF (NCT03047746) | Children's Hospital of Philadelphia | 2017-02-01 |
| Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT02605993) | Alexion Pharmaceuticals, Inc. | 2016-01-04 |
| Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. (NCT02588833) | Apellis Pharmaceuticals, Inc. | 2015-12-01 |
| Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT02534909) | Novartis Pharmaceuticals | 2015-09-09 |
| Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies (NCT02179359) | Masonic Cancer Center, University of Minnesota | 2014-09-02 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) (NCT01760096) | Institute of Hematology & Blood Diseases Hospital, China | 2013-01-01 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria (NCT01642979) | Institute of Hematology & Blood Diseases Hospital, China | 2012-07-01 |
| Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study (NCT01412047) | Alexion Pharmaceuticals, Inc. | 2012-03-01 |
| Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies (NCT01529827) | Roswell Park Cancer Institute | 2012-02-28 |
| Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease (NCT01294891) | Oregon Health and Science University | 2011-02-01 |
| Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders (NCT00856388) | Roswell Park Cancer Institute | 2009-01-14 |
| E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients (NCT01194804) | Alexion Pharmaceuticals, Inc. | 2008-04-01 |
| Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome (NCT00731328) | Asan Medical Center | 2008-04-01 |
| Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients (NCT01192399) | Alexion Pharmaceuticals, Inc. | 2007-11-01 |
| Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders (NCT00397813) | Fred Hutchinson Cancer Center | 2006-01-01 |
| Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies (NCT00143559) | St. Jude Children's Research Hospital | 2005-08-01 |
| Unrelated Donor Stem Cell Transplantation (NCT01364363) | Scripps Health | 2005-03-01 |
| Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry (NCT01374360) | Alexion Pharmaceuticals, Inc. | 2004-10-29 |
| Nonmyeloablative Allogeneic Transplant (NCT01272817) | Scripps Health | 2001-10-01 |
| Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT06028594) | Regeneron Pharmaceuticals | |
| Danicopan Early Access Program (NCT05982938) | Alexion Pharmaceuticals, Inc. | |