| Paroxysmal Nocturnal Hemoglobinuria |
ChiCTR-TRC-14004219: A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria |
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| Recruiting | N/A | 48 | | intravenous iron sucrose ;Oral iron dextran tablet | Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences; Level of the institution：, investigator-initiated research study | iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | | | | |
ChiCTR-ONC-13003994: Chemotherapy plus hematopoietic growth factors (DAG) for refractory paroxysmal nocturnal hemoglobinuria: diminishing PNH clone and stimulating hematopoisis |
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| Recruiting | N/A | 50 | | DA chemotherapy regimen plus G-CSF | Tianjin Medical University General Hospital; Tianjin Medical University General Hospital, self-financing | paroxymal nocturnal hemoglobinuria | | | | |
| Available | N/A | | NA | Pegcetacoplan | Apellis Pharmaceuticals, Inc. | C3G, IC-MPGN, C3 Glomerulopathy, C3 Glomerulonephritis, Complement 3 Glomerulopathy, Complement 3 Glomerulopathy (C3G), Complement 3 Glomerulonephritis, Dense Deposit Disease, DDD, Membranoproliferative Glomerulonephritis, Membranoproliferative Glomerulonephritis (MPGN), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | | | | |
NAP, NCT04671810: Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® |
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| Completed | N/A | 44 | RoW | Elizaria®, Eculizumab | AO GENERIUM | Paroxysmal Nocturnal Hemoglobinuria | 12/22 | 05/23 | | |
APPEX, NCT05842486: Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients |
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| Completed | N/A | 92 | Europe | Anti-C5 antibody treatment | Assistance Publique - Hôpitaux de Paris, Leeds Cancer Centre at St. James's University Hospital | Paroxysmal Nocturnal Hemoglobinuria | 03/23 | 03/23 | | |
NCT03329365: Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS |
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| Active, not recruiting | N/A | 200 | Canada | | Lawson Health Research Institute, Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Cerebral Vein Thrombosis | 03/23 | 03/24 | | |
| Available | N/A | | NA | Iptacopan, LNP023 | Novartis Pharmaceuticals | C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH) | | | | |
| Available | N/A | | NA | Danicopan, ALXN2040 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis | | | | |
NCT06028594: Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Available | N/A | | NA | Pozelimab, REGN3918, Cemdisiran, ALN-CC5 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | | | | |
| Available | N/A | | NA | Pozelimab, REGN3918 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH), CD55-deficient Protein-losing Enteropathy (PLE), CD59 Deficiency | | | | |
| Recruiting | N/A | 1000 | RoW | | AstraZeneca | Paroxysmal Nocturnal Hemoglobinuria | 11/25 | 11/25 | | |
NCT05274633: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Active, not recruiting | N/A | 120 | Europe | Ravulizumab, Ultomiris | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 06/24 | 02/25 | | |
NCT02179359: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies |
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| Recruiting | N/A | 25 | US | Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders | 01/25 | 08/25 | | |
NCT03047746: Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF |
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| Recruiting | N/A | 50 | US | CliniMACs | Children's Hospital of Philadelphia | Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes | 03/25 | 03/26 | | |
NCT06524726: The International PNH Interest Group PNH Registry |
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| Recruiting | N/A | 2000 | US | | International PNH Interest Group | Paroxysmal Nocturnal Hemoglobinuria | 05/29 | 05/29 | | |
| Recruiting | N/A | 128 | US | PNH-relevant therapies | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 05/25 | 05/25 | | |
NCT06312644: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy |
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| Recruiting | N/A | 75 | US | Ultomiris, There is no interventional drug. Ultomiris treatment is an inclusion criterion. | Alexion Pharmaceuticals, Inc. | Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), Neuromyelitis Optica Spectrum Disorder (NMOSD) | 11/33 | 11/33 | | |
NCT06606314: Specified Drug-use Surveillance of Fabhalta Capsules |
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| Not yet recruiting | N/A | 100 | NA | | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 09/28 | 09/28 | | |
NCT01374360: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry |
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| Recruiting | N/A | 5950 | US | | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 12/24 | 12/24 | | |
ChiCTR2300067949: The real world study of Eculizumab for paroxysmal nocturnal hemoglobinuria treatment in China |
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| Not yet recruiting | N/A | 10 | | Eculizumab | West China Hospitai, Sichuan University; West China Hospitai, Sichuan University, Apply for hospital project funds | paroxysmal nocturnal hemoglobinuria | | | | |
COMPLETE, NCT05776472: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Recruiting | N/A | 200 | Europe | Pegcetacoplan, Aspaveli, Empaveli | Swedish Orphan Biovitrum | Paroxysmal Nocturnal Hemoglobinuria | 01/26 | 07/27 | | |
| Recruiting | N/A | 500 | US | | Aplastic Anemia and MDS International Foundation, National Organization for Rare Disorders | Paroxysmal Nocturnal Hemoglobinuria | 05/26 | 05/31 | | |
