| Paroxysmal Nocturnal Hemoglobinuria |
NCT02946463 / 2016-002025-11: ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Completed | 3 | 272 | Europe, Canada, Japan, US, RoW | Ravulizumab, Eculizumab | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 02/23 | 02/23 | | |
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2018-003956-19: Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Not yet recruiting | 3 | 40 | Europe | Zilucoplan, RA101495, Solution for injection | Ra Pharmaceuticals, Inc., Ra Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Nocturnal Hemoglobinuria, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
NCT03748823 / 2017-002370-39: Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab |
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| Completed | 3 | 139 | Europe, Canada, US, RoW | Ravulizumab OBDS, Ravulizumab, ALXN1210 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 02/21 | 08/23 | | |
2018-004220-11: A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigational Drug APL-2 In Treating Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated with Anemia, In a Randomly Assigned Comparison with the Current Standard of Care Treatment Approved for PNH |
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| Not yet recruiting | 3 | 54 | Europe | APL-2, APL-2, Solution for injection/infusion | Apellis Pharmaceuticals, Inc., Apellis Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH), PNH is a rare disorder causing red blood cells to break down too early., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2020-004385-19: Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria |
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| Ongoing | 3 | 165 | Europe | iptacopan, LNP023, Capsule, hard, PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml, ACTHIB - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE 1 FLACONCINO POLVERE+1 SIRINGA PRERIEMPITA SOLVENTE 0.5 ML, MENVEO - POLV.E SOLUZ.PER SOLUZ. INIETT.-USO INTRAMUSCOLARE-COMP. CONIUG.LIOFILIZZ.MENA:FLAC.(VETRO) COMP. GONIUG.LIQUI.MENCWY:FLAC.(VETRO)-1 FLAC+1FLAC, BEXSERO - SOSPENSIONE INIETTABILE - USO INTRAMUSCOLARE - SIRINGA PRERIEMPITA (VETRO) - 0.5ML - 1 SIRINGA PRERIEMPITA CON AGO, Nimenrix, HIBERIX - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE PER USO INTRAMUSCOLARE 1 FLACONCINO DI POLVERE + 1 SIRINGA PRERIEMPITA DI SOLVENTE DA 0.5 ML | Novartis Pharma AG, NOVARTIS PHARMA AG, Novartis Farmacéutica, S.A., Novartis Pharma AG | Paroxysmal Nocturnal Hemoglobinuria (PNH), Blood disorder, Diseases [C] - Immune System Diseases [C20] | | | | |
COMMODORE 3, NCT04654468: A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition |
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| Active, not recruiting | 3 | 51 | RoW | Crovalimab | Hoffmann-La Roche | Paroxysmal Nocturnal Hemoglobinuria | 02/22 | 02/28 | | |
2019-000130-20: REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. |
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| Not yet recruiting | 3 | 147 | Europe, RoW | REGN3918, REGN3918, Lyophilisate for solution for injection, Solution for injection | Regeneron Pharmaceuticals, Inc., REGENERON PHARMACEUTICALS, INC., Regeneron Pharmaceuticals, Inc. | Paroxysmal nocturnal hemoglobinuria, Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by the destruction of red blood cells., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
ALPHA, NCT04469465 / 2019-003829-18: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) |
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| Completed | 3 | 86 | Europe, Canada, Japan, US, RoW | Danicopan, ALXN2040, Placebo, C5 Inhibitor | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 06/22 | 01/24 | | |
| Completed | 3 | 97 | Europe, Japan, US, RoW | LNP023, iptacopan, Eculizumab, Ravulizumab | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 09/22 | 03/23 | | |
| Completed | 3 | 40 | Europe, RoW | Iptacopan (LNP023), Iptacopan | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 11/22 | 04/23 | | |
COMMODORE 2, NCT04434092 / 2019-004931-21: A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. |
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| Active, not recruiting | 3 | 204 | Europe, Japan, RoW | Crovalimab, Eculizumab | Hoffmann-La Roche, Chugai Pharmaceutical | Paroxysmal Nocturnal Hemoglobinuria | 11/22 | 06/28 | | |
2021-004931-10: A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal Hemoglobinuria |
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| Ongoing | 3 | 288 | Europe | Pozelimab, Cemdisiran, REGN3918, ALN-CC5, Solution for injection, Solution for injection/infusion | Regeneron Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc. | Paroxysmal nocturnal hemoglobinuria (PNH), Paroxysmal nocturnal hemoglobinuria (PNH), Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
ACCESS 2, NCT05131204 / 2020-002761-33: Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Terminated | 3 | 3 | US | Cemdisiran, ALN-CC5, Eculizumab, Soliris, Pozelimab, REGN3918, Ravulizumab, ALXN1210, Ultomiris | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 07/23 | 07/23 | | |
NCT06449001: Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis |
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| Not yet recruiting | 3 | 6 | NA | Danicopan | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis | 01/27 | 01/28 | | |
HRS-5965-301, NCT06593938: A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy |
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| Not yet recruiting | 3 | 70 | RoW | HRS-5965 capsule, Eculizumab Injection | Chengdu Suncadia Medicine Co., Ltd. | Paroxysmal Nocturnal Hemoglobinuria | 12/25 | 12/25 | | |
NCT06578949: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Not yet recruiting | 3 | 18 | RoW | Ravulizumab, ALXN1210 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, PNH | 10/25 | 09/26 | | |
APPULSE, NCT05630001: Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. |
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| Active, not recruiting | 3 | 52 | Europe, US, RoW | Iptacopan | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 10/24 | 10/24 | | |
Soliris, NCT05886244: Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China |
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| Active, not recruiting | 3 | 25 | RoW | Eculizumab | Alexion Pharmaceuticals, Inc., AstraZeneca | Paroxysmal Nocturnal Hemoglobinuria | 06/25 | 06/25 | | |
| Active, not recruiting | 3 | 160 | Europe, Canada, Japan, US, RoW | Pegcetacoplan | Apellis Pharmaceuticals, Inc. | PNH | 06/25 | 06/25 | | |
ChiCTR2400088580: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Not yet recruiting | 3 | 18 | | Ravulizumab | Peking Union Medical College Hospital; Peking Union Medical College Hospital, Astrazeneca Global R&D (China) Co., LTD | PNH | | | | |
| Active, not recruiting | 3 | 80 | Europe, Canada, Japan, US, RoW | Danicopan, ALXN2040, ACH-0144471 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 01/27 | 01/27 | | |
ACCESS-1, NCT05133531 / 2020-004486-40: A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment |
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| Recruiting | 3 | 190 | Europe, Canada, Japan, US, RoW | Ravulizumab, ALXN1210, Ultomiris, Pozelimab, REGN3918, Cemdisiran, ALN-CC5, Eculizumab, Soliris | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 04/27 | 04/27 | | |
NCT04747613: Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Recruiting | 3 | 250 | Europe, Japan, US, RoW | Iptacopan, LNP023 | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 01/28 | 01/28 | | |
ACCESS-EXT, NCT05744921 / 2021-004931-10: A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works. |
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| Recruiting | 3 | 202 | Europe, Canada, Japan, RoW | Pozelimab, REGN3918, Cemdisiran, ALN-CC5 | Regeneron Pharmaceuticals, Regeneron Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 02/29 | 02/29 | | |
COMMODORE 1, NCT04432584 / 2020-000597-26: A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors |
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| Active, not recruiting | 3 | 190 | Europe, Canada, Japan, US, RoW | Crovalimab, Eculizumab | Hoffmann-La Roche, Chugai Pharmaceutical | Paroxysmal Nocturnal Hemoglobinuria | 09/29 | 09/29 | | |
