10 recruiting, 9 completed, 4 active not recruiting, 2 not yet recruiting. 14 Phase 3, 7 Phase 2, 2 Not applicable, 1 Phase 1.
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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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy iptacopan - Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplantation (HSCT) | Not applicable | Recruiting | First Affiliated Hospital of Zhejiang University | 30 | 2026-08-25 | 2026-08-26 |
| CHART-C3G/CLNP023B12011 Iptacopan - C3 Glomerulopathy, Complement-mediated Kidney Disease | Not yet recruiting | Novartis Pharmaceuticals | 83 | 2026-01-01 | 2026-01-09 | |
| Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN iptacopan - Primary Immunoglobulin A Nephropathy (IgAN) | Phase 3 | Recruiting | Novartis Pharmaceuticals | 31 | 2025-11-27 | 2026-09-09 |
| Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients LNP023 - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | Recruiting | Novartis Pharmaceuticals | 12 | 2025-10-28 | 2026-08-07 |
| Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry Iptacopan - Hemoglobinuria, Paroxysmal | Active not recruiting | Novartis Pharmaceuticals | 200 | 2025-03-31 | 2026-06-23 | |
| A Mobile App-Based Study to Evaluate Disease Burden and Treatment Patterns in Immunoglobulin A Nephropathy (IgAN) in the US No intervention, Other treatment, Iptacopan - Immunoglobulin A Nephropathy | Recruiting | Novartis Pharmaceuticals | 300 | 2025-03-04 | 2025-06-17 | |
| A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA) Iptacopan - AIHA - Warm Autoimmune Hemolytic Anemia, AIHA - Cold Autoimmune Hemolytic Anemia | Not applicable | Not yet recruiting | Bing Han | 20 | 2025-03-01 | 2025-02-26 |
| Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN Iptacopan - Glomerulonephritis, IGA, Immunoglobulin A Nephropathy | Phase 2 | Recruiting | Novartis Pharmaceuticals | 20 | 2025-02-21 | 2026-07-29 |
| Iptacopan in Patients With ANCA Associated Vasculitis Iptacopan, Placebo, Rituximab - Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis | Phase 2 | Active not recruiting | Novartis Pharmaceuticals | 84 | 2024-08-05 | 2026-10-15 |
| A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG. Iptacopan, Matching Placebo - Generalized Myasthenia Gravis | Phase 3 | Active not recruiting | Novartis Pharmaceuticals | 146 | 2024-07-31 | 2026-08-12 |
| Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Iptacopan - Atypical Hemolytic Uremic Syndrome | Phase 3 | Recruiting | Novartis Pharmaceuticals | 50 | 2024-02-28 | 2026-04-29 |
| Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN Placebo, iptacopan - IC-MPGN | Phase 3 | Recruiting | Novartis Pharmaceuticals | 106 | 2023-10-02 | 2026-07-13 |
| Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. Iptacopan - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Completed | Novartis Pharmaceuticals | 52 | 2023-04-24 | 2026-01-13 |
| Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V Iptacopan (part 1), Iptacopan (part 2), Placebo + standard of care - Lupus Nephritis | Phase 2 | Terminated | Novartis Pharmaceuticals | 81 | 2022-08-10 | 2026-10-15 |
| A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration Iptacopan (LNP023), Placebo - Age-Related Macular Degeneration | Phase 2 | Completed | Novartis Pharmaceuticals | 168 | 2022-02-17 | 2026-10-27 |
| Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy Iptacopan - Atypical Hemolytic Uremic Syndrome | Phase 3 | Completed | Novartis Pharmaceuticals | 34 | 2022-01-17 | 2026-08-07 |
| Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders Iptacopan - Immune Thrombocytopenia (ITP), Cold Agglutinin Disease (CAD) | Phase 2 | Terminated | Novartis Pharmaceuticals | 19 | 2021-12-21 | 2025-10-16 |
| Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to Matched Control Healthy Participants With Normal Hepatic Function. Iptacopan - Hepatic Impairment | Phase 1 | Completed | Novartis Pharmaceuticals | 38 | 2021-11-10 | 2023-07-24 |
| A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy LNP023 - Primary IgA Nephropathy | Phase 3 | Recruiting | Novartis Pharmaceuticals | 540 | 2021-09-20 | 2026-10-05 |
| Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. Placebo, iptacopan - C3G | Phase 3 | Recruiting | Novartis Pharmaceuticals | 98 | 2021-07-28 | 2026-07-13 |
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Hemoglobinuria, Paroxysmal (9) · Proteinuria (6) · Glomerulonephritis, IGA (5) · Anemia (4) · Thrombotic Microangiopathies (3) · Anemia, Hemolytic, Autoimmune (2) · Atypical Hemolytic Uremic Syndrome (2) · Hemolysis (2) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis (1) · Geographic Atrophy (1) · Glomerulonephritis (1) · Glomerulonephritis, Membranoproliferative (1) · Granulomatosis with Polyangiitis (1) · Lupus Erythematosus, Systemic (1) · Lupus Nephritis (1)
Novartis Pharmaceuticals (26) · Bing Han (1) · First Affiliated Hospital of Zhejiang University (1)
United States (19) · Germany (16) · China (15) · United Kingdom (15) · France (14) · Italy (14) · Spain (13) · Brazil (11) · Japan (11) · South Korea (11) · Argentina (10) · Turkey (Türkiye) (10) · Czechia (9) · Israel (8) · Netherlands (8)
PMID 40694294, PMID 38477987, PMID 40447351, PMID 33765419, PMID 35561315, PMID 35675911, PMID 37314061, PMID 37528520, PMID 37772889, PMID 37914086, PMID 38299639, PMID 39453772, PMID 40632630, PMID 41016405, PMID 41366634, PMID 41910396
2023 (4), 2017 (1), 2018 (1), 2019 (1)