55 completed, 16 active not recruiting, 13 recruiting, 8 terminated. 32 Phase 2, 31 Phase 3, 13 Phase 1, 6 Not applicable, 2 Phase 4.
Follow Paroxysmal Nocturnal Hemoglobinuria: new trials by email Literature Review of Paroxysmal Nocturnal Hemoglobinuria trials Research Report on Paroxysmal Nocturnal Hemoglobinuria
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 |
| 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 |
| 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 |
| 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 |
| 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 | |
| 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 |
| A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients NTQ5082 capsules 200 mg, Eculizumab Injection - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Nanjing Chia-tai Tianqing Pharmaceutical | 78 | 2025-11-01 | 2025-09-17 |
| Long-term Safety of Danicopan: IPIG Registry-based Cohort Study Danicopan - Paroxysmal Nocturnal Hemoglobinuria, PNH | Active not recruiting | Alexion Pharmaceuticals, Inc. | 50 | 2025-08-18 | 2026-02-17 | |
| A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria XH-S003 Capsule - Paroxysmal Nocturnal Hemoglobinuria | Not applicable | Enrolling by invitation | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 26 | 2025-08-15 | 2026-09-25 |
| Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis Danicopan - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 3 | Recruiting | Alexion Pharmaceuticals, Inc. | 6 | 2025-08-11 | 2025-12-23 |
| NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice Paroxysmal Nocturnal Hemoglobinuria | Terminated | Novartis Pharmaceuticals | 6 | 2025-07-29 | 2026-08-17 | |
| A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects HRS-5965 Capsules - IgA Nephropathy, Paroxysmal Nocturnal Hemoglobinuria | Phase 1 | Completed | Chengdu Suncadia Medicine Co., Ltd. | 16 | 2025-06-30 | 2025-08-20 |
| A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China LNP023 - Paroxysmal Nocturnal Hemoglobinuria | Recruiting | Novartis Pharmaceuticals | 80 | 2025-06-10 | 2026-06-01 | |
| A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients NTQ5082 100mg qd, NTQ5082 200mg qd - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Active not recruiting | Nanjing Chia-tai Tianqing Pharmaceutical | 24 | 2025-01-23 | 2025-09-23 |
| Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment HRS-5965 capsule - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Completed | Chengdu Suncadia Medicine Co., Ltd. | 39 | 2025-01-07 | 2026-01-08 |
| Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria HSK39297 tablets - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Recruiting | Haisco Pharmaceutical Group Co., Ltd. | 47 | 2025-01-03 | 2024-12-20 |
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Hemoglobinuria, Paroxysmal (105) · Anemia, Aplastic (10) · Anemia (8) · Hemolysis (8) · Leukemia, Myeloid, Acute (7) · Hemoglobinuria (6) · Lymphoma, Non-Hodgkin (6) · Myelodysplastic Syndromes (6) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (6) · Hematologic Diseases (5) · Hodgkin Disease (5) · Leukemia, Lymphocytic, Chronic, B-Cell (5) · Leukemia, Myelomonocytic, Chronic (5) · Multiple Myeloma (5) · Anemia, Diamond-Blackfan (4)
Hemic and Lymphatic Diseases (105) · Bone Marrow Diseases (103) · Anemia, Hemolytic (101) · Hematologic Diseases (100) · Myelodysplastic Syndromes (99) · Anemia (97) · Pathological Conditions, Signs and Symptoms (21) · Pathologic Processes (17) · Bone Marrow Failure Disorders (10) · Disease Attributes (9) · Female Urogenital Diseases (9) · Female Urogenital Diseases and Pregnancy Complications (9) · Male Urogenital Diseases (9) · Neoplasms by Histologic Type (9) · Urogenital Diseases (9)
eculizumab (20) · ravulizumab (10) · danicopan (7) · Whole-Body Irradiation (5) · fludarabine phosphate (5) · Peripheral Blood Stem Cell Transplantation (4) · Cyclosporine (3) · Mycophenolic Acid (3) · Tacrolimus (3) · iptacopan (3) · pegcetacoplan (3) · thymoglobulin (3) · Antilymphocyte Serum (2) · Cyclophosphamide (2) · Glucocorticoids (2)
Alexion Pharmaceuticals, Inc. (18) · Novartis Pharmaceuticals (9) · Regeneron Pharmaceuticals (8) · Wuhan Createrna Science and Technology Co., Ltd (6) · Chengdu Suncadia Medicine Co., Ltd. (5) · BioCryst Pharmaceuticals (4) · AO GENERIUM (3) · Apellis Pharmaceuticals, Inc. (3) · Hoffmann-La Roche (3) · Nanjing Chia-tai Tianqing Pharmaceutical (3) · Omeros Corporation (3) · Roswell Park Cancer Institute (3) · AstraZeneca (2) · Fred Hutchinson Cancer Center (2) · Institute of Hematology & Blood Diseases Hospital, China (2)
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George Chen (2) · Oksana Markova, MD (2) · Alice Houk, MS (1) · Antionio Risitano (1) · Ashish Gupta, MBBS, MPH (1) · B. Smith (1) · Beatrice Drexler, Dr. med (1) · Brenda Sandmaier (1) · Camille Bedrosian, MD (1)
PMID 39079163, PMID 39535306, PMID 38727860, PMID 40720060, PMID 40978458, PMID 21222185, PMID 23934275, PMID 26352814, PMID 27766064, PMID 30171081, PMID 30630975, PMID 32499241, PMID 33730455, PMID 34398258, PMID 34445916, PMID 34922648, PMID 35561315, PMID 35796199, PMID 35869170, PMID 35869984, PMID 36055332, PMID 36108594, PMID 36272026, PMID 36326854, PMID 36750531, PMID 36848639, PMID 37052120, PMID 37199789, PMID 37421604, PMID 37655691, PMID 37707673, PMID 38030318, PMID 38551806, PMID 38593241, PMID 38884175, PMID 38924124, PMID 39171864, PMID 39700502, PMID 40285403, PMID 40395624, PMID 40560515, PMID 40694294, PMID 40708708, PMID 41124654, PMID 41566054, PMID 41795103, PMID 42287326, PMID 42707331
2019 (5), 2023 (5), 2017 (3), 2014 (1), 2015 (1), P01CA015396 (1), P01CA018029 (1), P30CA015704 (1), U10HL069294 (1)