| Paroxysmal Nocturnal Hemoglobinuria |
| Recruiting | 1b | 10 | RoW | OMS906 study drug | Omeros Corporation | Paroxysmal Nocturnal Hemoglobinuria | 10/23 | 06/24 | | |
NCT05972967: Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab |
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| Recruiting | 1b | 12 | Europe | OMS906 Study Drug - 3 mg/kg, OMS906 Study Drug - 5 mg/kg, Ravulizumab | Omeros Corporation | Paroxysmal Nocturnal Hemoglobinuria | 05/25 | 07/25 | | |
ACTRN12615001143516: A first in human study assessing the safety and pharmacokinetics (blood levels) of RA101495. |
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| Recruiting | 1 | 40 | Rest Of World | RA101495 | Ra Pharmaceuticals, Inc., Ra Pharmaceuticals, Inc. | Paroxysmal nocturnal hemoglobinuria | | | | |
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NCT06326814: A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults |
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| Completed | 1 | 54 | US | Humanized anti-Factor Bb monoclonal antibody, SAR443809, Placebo | Sanofi | Paroxysmal Nocturnal Hemoglobinuria | 05/23 | 05/23 | | |
ACTRN12621000041853: A Phase 1 first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of KP104. |
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| Completed | 1 | 64 | | | Kira Pharmaceuticals, Kira Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH) | | | | |
NCT05490017: To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 |
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| Completed | 1 | 80 | RoW | KP104, Placebo | Kira Pharmacenticals (US), LLC. | Paroxysmal Nocturnal Hemoglobinuria | 05/22 | 09/22 | | |
NCT05828485: Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects |
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| Completed | 1 | 12 | RoW | MY008211A tablets, no other name | Wuhan Createrna Science and Technology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria | 10/22 | 10/22 | | |
NCT05642585: A Study of Single-dose MY008211A in Healthy Adults |
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| Completed | 1 | 63 | RoW | MY008211A tablets, no name, Placebo | Wuhan Createrna Science and Technology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria | 06/23 | 06/23 | | |
NCT05828472: A Study of Multiple Ascending Doses MY008211A in Healthy Adults |
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| Completed | 1 | 40 | RoW | MY008211A tablets, no other name, MY008211A tablets matched placebo, placebo | Wuhan Createrna Science and Technology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria | 06/23 | 06/23 | | |
NCT06294301: A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants |
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| Recruiting | 1 | 78 | RoW | LP-005 Dose 1 (Single), LP-005 Dose 2 (Single), LP-005 Dose 3 (Single), LP-005 Dose 4 (Single), LP-005 Dose 5 (Single), LP-005 Dose 6 (Single), Placebo (Single), LP-005 Dose 7 (Multiple), LP-005 Dose 8 (Multiple), LP-005 Dose 9 (Multiple), Placebo (Multiple) | Longbio Pharma | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 04/24 | 10/24 | | |
ChiCTR2300072738: Single-centre, double-blind, placebo-controlled, dose-escalation phase Ia clinical study to assess the safety, tolerability and pharmacokinetic/pharmacodynamic profiles of EA5 in a single dose in adult healthy subjects |
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| Not yet recruiting | 1 | 28 | | 90 mg test drug ;180 mg test drug ;360 mg test drug vs placebo ;720 mg test drug vs placebo ;1440 mg test drug vs placebo | The Second Affiliated Hospital of Anhui Medical University; Suzhou Bowei Biotechnology Co., Ltd, Suzhou Bowei Biotechnology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria, PNH | | | | |
NCT05083364: Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease |
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| Recruiting | 1 | 60 | Europe, RoW | ARO-C3, Placebo | Arrowhead Pharmaceuticals | C3 Glomerulopathy, IgA Nephropathy | 12/24 | 06/25 | | |
NCT06490991: Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects |
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| Active, not recruiting | 1 | 16 | RoW | HRS-5965 tablets；HRS-5965 capsules | Chengdu Suncadia Medicine Co., Ltd. | Paroxysmal Nocturnal Hemoglobinuria，IgA Nephropathy | 08/24 | 12/24 | | |
NCT06543459: To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects |
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| Not yet recruiting | 1 | 16 | RoW | MY008211A tablets, Placebo | Wuhan Createrna Science and Technology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria | 09/24 | 12/24 | | |
NCT06100900: Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria |
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| Recruiting | 1 | 15 | RoW | BCX10013 | BioCryst Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 08/24 | 08/24 | | |
PNH, NCT05876312: Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria () Patients |
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| Recruiting | 1 | 53 | RoW | ADX-038, siRNA, Placebo, Saline | ADARx Pharmaceuticals, Inc., ADARx Australia Pty Ltd | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 11/24 | 06/25 | | |
