100 completed, 30 recruiting, 21 active not recruiting, 13 not yet recruiting. 70 Phase 2, 49 Phase 3, 28 Phase 1, 6 Not applicable, 3 Phase 4.
Follow Hemoglobinuria, Paroxysmal: new trials by email Literature Review of Hemoglobinuria, Paroxysmal trials Research Report on Hemoglobinuria, Paroxysmal
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris NM8074, Soliris - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Not yet recruiting | NovelMed Therapeutics | 12 | 2027-01-01 | 2026-04-13 |
| A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH. FXS6837 Capsule, Eculizumab infusion - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 90 | 2026-12-10 | 2026-08-05 |
| Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria SGB-9768 - PNH - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Not yet recruiting | Suzhou Sanegene Bio Inc. | 24 | 2026-10-31 | 2026-09-04 |
| A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy VSA012, Eculizumab (Soliris®) - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 3 | Not yet recruiting | Bisirna Therapeutics (Suzhou) Co., Ltd. | 70 | 2026-10-29 | 2026-09-11 |
| Efficacy and Safety of Lanoracopan in the Treatment of PNH Lanoracopan Hydrochloride - Paroxysmal Nocturnal Hemoglobinuria, PNH | Not yet recruiting | Peking Union Medical College Hospital | 90 | 2026-09-01 | 2026-08-26 | |
| To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants. Part1: TUL321, Part1: Placebo, Part2: TUL321 - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 1 | Recruiting | Zhuhai United Laboratories Co., Ltd. | 94 | 2026-08-26 | 2026-09-01 |
| A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy | Recruiting | Novartis Pharmaceuticals | 21 | 2026-06-15 | 2026-06-26 | |
| A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Recruiting | Regeneron Pharmaceuticals | 35 | 2026-06-11 | 2026-08-05 |
| A Long-term Efficacy and Safety of NTQ5082 Capsules NTQ5082 capsules 200 mg - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Nanjing Chia-tai Tianqing Pharmaceutical | 78 | 2026-06-01 | 2025-09-17 |
| Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NM8074 - PNH - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Not yet recruiting | NovelMed Therapeutics | 6 | 2026-06-01 | 2025-03-30 |
| Danicopan PMS in Korea Paroxysmal Nocturnal Hemoglobinuria | Recruiting | AstraZeneca | 27 | 2026-04-10 | 2026-10-13 | |
| Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy NM8074 - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Not yet recruiting | NovelMed Therapeutics | 12 | 2026-04-01 | 2025-03-11 |
| A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants CMS-D017 Capsule, CMS-D017 Placebo Capsule - Paroxysmal Nocturnal Hemoglobinuria, PNH, Complement-mediated Kidney Disease | Phase 1 | Recruiting | Shenzhen Kangzhe Biotechnology Co., Ltd. | 88 | 2026-03-05 | 2026-04-08 |
| Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria HS-10542 - Paroxysmal Nocturnal Hemoglobinuria | Phase 1 / Phase 2 | Recruiting | Jiangsu Hansoh Pharmaceutical Co., Ltd. | 50 | 2026-02-14 | 2026-04-13 |
| A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor ALN-CFB, Placebo - Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia | Phase 1 / Phase 2 | Recruiting | Regeneron Pharmaceuticals | 24 | 2026-02-11 | 2026-07-09 |
| SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China SLN12140 - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | Not yet recruiting | Linno Pharmaceuticals, Inc. | 10 | 2026-02-10 | 2026-02-04 |
| Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data Danicopan, Soliris/Ultomiris - Paroxysmal Nocturnal Hemoglobinuria, PNH | Active not recruiting | Alexion Pharmaceuticals, Inc. | 50 | 2026-01-14 | 2026-02-17 | |
| REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH) | Recruiting | Novartis Pharmaceuticals | 50 | 2026-01-13 | 2026-06-25 | |
| Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) LP-005 Injection - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Not yet recruiting | Longbio Pharma | 30 | 2025-12-05 | 2025-12-05 |
| Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) MY008211A tablets - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | Not yet recruiting | Wuhan Createrna Science and Technology Co., Ltd | 40 | 2025-12-01 | 2025-07-29 |
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