Table 1.
List of compounds under development targeting the complement system.
| Drug | Indication | Trial Phase/Study Type | Recruitment Status | NCT Number |
|---|---|---|---|---|
| Eculizumab, C5-targeting antibody | PNH | Phase 2/3 | Only recruiting Trials are listed |
NCT04079257 |
| aHUS-associated multiple organ dysfunction syndrome in hematopoietic stem cell transplant recipients | Phase 2 | NCT03518203 | ||
| Refractory GMG | Phase 3 | NCT03759366 | ||
| COVID-19 | Phase 2 | NCT04346797 | ||
| CHAPLE disease | Prospective Cohort | NCT03950804 | ||
| PNH | Phase 3 | NCT04432584 | ||
| PNH | Phase 3 | NCT03818607 | ||
| PNH | Phase 3 | NCT04434092 | ||
| End Stage Liver Disease | Phase 1 | NCT03468140 | ||
| PNH | Observational Study | NCT01374360 | ||
| aHUS | Observational Study | NCT01522183 | ||
| Neuromyelitis optica spectrum disorder | Phase 2/3 | NCT04155424 | ||
| ABP959, C5-targeting antibody (eculizumab biosimilar) | PNH | Phase 3 | Recruiting | NCT03818607 |
| SB12 C5-targeting antibody (eculizumab biosimilar) | PNH | Phase 3 | Active, not recruiting | NCT04058158 |
| BCD-148, C5-targeting antibody (eculizumab biosimilar) | PNH | Phase 3 | Active, not recruiting | NCT04060264 |
| Healthy subjects | Phase 1 | Completed | NCT04027803 | |
| Nomacopan, C5-targeting protein | PNH, aHUS | Phase 3 | Recruiting | NCT03829449 |
| BP | Phase 2 | Completed | NCT04035733 | |
| PNH | Phase 3 | Completed | NCT03588026 | |
| PNH | Phase 2 | Enrolling by invitation | NCT03427060 | |
| PNH | Phase 2 | Completed | NCT02591862 | |
| AKC | Phase 1/2 | Active, not recruiting | NCT04037891 | |
| Ravulizumab, C5-targeting antibody | COVID-19, thrombotic microangiopathies acute kidney injury | Phase 3 | Only recruiting Trials are listed |
NCT04570397 |
| Neuromyelitis optica Neuromyelitis optica spectrum disorder | Phase 3 | NCT04201262 | ||
| TMA | Phase 3 | NCT04557735 | ||
| ALS | Phase 3 | NCT04248465 | ||
| COVID-19 severe pneumonia, acute lung injury, acute respiratory distress syndrome pneumonia, Viral | Phase 3 | NCT04369469 | ||
| TMA | Phase 3 | NCT04543591 | ||
| PNH | Phase 3 | NCT03406507 | ||
| COVID-19 | Phase 4 | NCT04390464 | ||
| Atypical hemolytic-uremic syndrome | Observational Study | NCT01522183 | ||
| PNH | Observational Study | NCT01374360 | ||
| PNH | Phase 3 | NCT04432584 | ||
| Pozelimab, C5-targeting antibody | Healthy volunteer | Phase 1 | Active, not recruiting | NCT04491838 |
| Healthy subjects (in combination with cemdisiran) | Phase 1 | Recruiting | NCT04601844 | |
| CHAPLE | Phase 2/3 | Recruiting | NCT04209634 | |
| Crovalimab, C5-targeting antibody | PNH | Phase 3 | Not yet recruiting | NCT04654468 |
| PNH | Phase 3 | Recruiting | NCT04432584 | |
| PNH | Phase 3 | Recruiting | NCT04434092 | |
| PNH | Phase 1/2 | Active, not recruiting | NCT03157635 | |
| Cemdisiran, C5 targeting RNAi therapeutic | IgAN, Berger disease, glomerulonephritis | Phase 2 | Recruiting | NCT03841448 |
| Healthy | Phase 1 | Recruiting | NCT04601844 | |
| aHUS | Phase 2 | Withdrawn | NCT03303313 | |
| PNH | Phase 1 | Completed | NCT02352493 | |
| TMA | Phase2 | Not yet recruiting | NCT03999840 | |
| IFX-1, C5a targeting antibody | Pyoderma gangrenosum | Phase 2 | Recruiting | NCT03971643 |
| Severe COVID-19 pneumonia | Phase 2/3 | Recruiting | NCT04333420 | |
| GPA, MPA | Phase 2 | Active, not recruiting | NCT03712345 | |
| GPA, MPA | Phase 2 | Active, not recruiting | NCT03895801 | |
| HS | Phase 2 | Completed | NCT03487276 | |
| HS | Phase 2 | Completed | NCT03001622 | |
| Systemic inflammatory response syndrome, C.surgical procedure, cardiac | Phase 2 | Completed | NCT02866825 | |
| Severe sepsis, septic shock | Phase 2 | Completed | NCT02246595 | |
| Drug Safety | Phase 1 | Completed | NCT01319903 | |
| BDB-001, C5a targeting antibody | COVID-19 pneumonia | Phase 2/3 | Recruiting | NCT04449588 |
| Solid tumor | Phase 1 | Recruiting | NCT04196530 | |
| Solid tumor | Phase 1 | Recruiting | NCT03486301 | |
| Solid tumor, pancreatic cancer, virus associated tumors, non-small cell lung cancer, melanoma, bladdder cancer, triple negative breast cancer | Phase 2 | Not yet recruiting | NCT03915678 | |
| ALXN1007, C5a targeting antibody | Antiphospholipid (aPL)-positive | Phase 2 | Terminated | NCT02128269 |
| Acute (GVHD), GIGVHD | Phase 2 | Terminated | NCT02245412 | |
| Healthy | Phase 1 | Completed | NCT01883544 | |
| Healthy | Phase 1 | Completed | NCT01454986 | |
| Zilucoplan, C5-targeting peptide | GMG | Phase 3 | Recruiting | NCT04115293 |
| ALS | Phase 2/3 | Enrolling by invitation | NCT04436497 | |
| ALS | Phase 2/3 | Recruiting | NCT04297683 | |
| GMG | Phase 3 | Recruiting | NCT04225871 | |
| COVID-19 | Phase 2 | Active, not recruiting | NCT04382755 | |
| COVID-19 | Phase 3 | Recruiting | NCT04590586 | |
| IMNM | Phase 2 | Recruiting | NCT04025632 | |
| PNH | Phase 2 | Active, not recruiting | NCT03225287 | |
| PNH | Phase 2 | Completed | NCT03030183 | |
| PNH | Phase 2 | Completed | NCT03078582 | |
| GMG | Phase 2 | Completed | NCT03315130 | |
| Tesidolumab, C5-targeting protein | Wet AMD, Exudative MD | Phase 2 | Completed | NCT01535950 |
| PNH | Phase 2 | Active, not recruiting | NCT02534909 | |
| Transplant associate microangiopathy | Phase 2 | Terminated | NCT02763644 | |
| Kidney transplantation | Phase 1 | Completed | NCT02878616 | |
| Geographic atrophy, AMD | Phase 2 | Completed | NCT01527500 | |
| Advanced AMD | Phase 1 | Completed | NCT01255462 | |
| Geographic atrophy | Phase 1 | Completed | NCT02515942 | |
| Neovascular AMD | Phase 2 | Terminated | NCT01624636 | |
| Non-infectious intermediate uveitis, non-infectious posterior uveitis, non-infectious panuveitis | Phase 2 | Completed | NCT01526889 | |
| Avacopan, C5aR1-targeting small molecule | ANCA-associated vasculitis | Phase 3 | Completed | NCT02994927 |
| ANCA-associated vasculitis | Phase 2 | Completed | NCT02222155 | |
| Vasculitis | Phase 2 | Completed | NCT01363388 | |
| aHUS | Phase 2 | Terminated | NCT02464891 | |
| IgAN | Phase 2 | Completed | NCT02384317 | |
| Hidradenitis, suppurativa acne inversa | Phase 2 | Active, not recruiting | NCT03852472 | |
| C3 Glomerulopathy | Phase 3 | Recruiting | NCT03301467 | |
| Avacincaptad, C5-targeting oligonucleotide | Geographic atrophy secondary to AMD | Phase 2/3 | Completed | NCT02686658 |
| IPCV | Phase 2 | Completed | NCT02397954 | |
| AMD | Phase 2 | Completed | NCT03362190 | |
| IPCV | Phase 2 | Withdrawn | NCT03374670 | |
| Stargardt disease 1 | Phase 2 | Recruiting | NCT03364153 | |
| Geographic atrophysecondary to MD | Phase 3 | Recruiting | NCT04435366 | |
| Avdoralimab, C5aR1-targeting antibody | BP | Phase 2 | Recruiting | NCT04563923 |
| COVID-19, infection advanced and metastatic hematological or solid tumor | Phase 2 | Recruiting | NCT04333914 | |
| COVID-19 | Phase 2 | Active, not recruiting | NCT04371367 | |
| Advanced solid tumors | Phase 1 | Recruiting | NCT03665129 |
Abbreviations: NCT, national clinical trial; aHUS, atypical hemolytic uremic syndrome; AKC, atopic keratoconjunctivitis; ALS, amyotrophic lateral sclerosis; AMD, age-related macular degeneration; ANCA, anti-neutrophil cytoplasmic autoantibody; BP, Bullous pemphigoid; CHAPLE, complement hyperactivation, angiopathic thrombosis, protein losing enteropathy; COVID-19, coronavirus disease 2019; GBS, Guillain-Barré syndrome; GIGVHD, gastrointestinal graft versus host disease; GMG, generalized myasthenia gravis; GPA, granulomatosis with polyangiitis IgAN, IgA nephropathy; GVHD, graft versus host disease; HS, hidradenitis suppurativa; IMNM, immune-mediated necrotizing myopathy; IPCV, idiopathic polypoidal choroidal vasculopathy; MD, macular degeneration; MPA, microscopic polyangiitis; PNH, paroxysmal nocturnal hemoglobinuria; TMA, complement-mediated thrombotic microangiopathy.