Pegcetacoplan acetate has been evaluated in numerous clinical trials spanning Phases 1 through 4, investigating complement-mediated and oncological indications such as geographic atrophy, paroxysmal nocturnal hemoglobinuria, complement 3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, amyotrophic lateral sclerosis, and recurrent ovarian or fallopian tube carcinomas. Sponsored by pharmaceutical developers (including Apellis Pharmaceuticals, Swedish Orphan Biovitrum, and Boehringer Ingelheim) and academic centers (such as Columbia University and Roswell Park Cancer Institute), these trials encompass statuses including recruiting, active not recruiting, completed, approved for marketing, and terminated.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07746895 | RECRUITING | C3 Glomerulopathy (C3G); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) |
Swedish Orphan Biovitrum |
2026-07-09 | PHASE4 |
| NCT07020832 | WITHDRAWN | Delayed Graft Function; End Stage Renal Disease; Deceased Donor Kidney Transplant |
Apellis Pharmaceuticals, Inc. |
2026-05-18 | PHASE3 |
| NCT04901936 | RECRUITING | Paroxysmal Nocturnal Hemoglobinuria (PNH); Paroxysmal Hemoglobinuria |
Apellis Pharmaceuticals, Inc. |
2021-02-04 | PHASE2 |
| NCT04919629 | RECRUITING | Fallopian Tube Carcinosarcoma; Fallopian Tube Clear Cell Adenocarcinoma; Fallopian Tube Endometrioid Adenocarcinoma; Fallopian Tube Serous Adenocarcinoma; Ovarian Carcinosarcoma; Ovarian Clear Cell Adenocarcinoma; Ovarian Endometrioid Adenocarcinoma; Ovarian Serous Adenocarcinoma; Primary Peritoneal Carcinosarcoma; Primary Peritoneal Clear Cell Adenocarcinoma; Primary Peritoneal Endometrioid Adenocarcinoma; Primary Peritoneal Serous Adenocarcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma |
Roswell Park Cancer Institute |
2023-04-27 | PHASE2 |
| NCT07214298 | RECRUITING | Metastatic Pancreatic Ductal Adenocarcinoma; Stage IV Pancreatic Cancer AJCC v8 |
Roswell Park Cancer Institute |
2026-02-16 | PHASE1; PHASE2 |
| NCT05809531 | ACTIVE_NOT_RECRUITING | C3G; IC-MPGN; C3 Glomerulopathy; C3 Glomerulonephritis; Complement 3 Glomerulopathy; Complement 3 Glomerulopathy (C3G); Complement 3 Glomerulonephritis; Dense Deposit Disease; DDD; Membranoproliferative Glomerulonephritis; Membranoproliferative Glomerulonephritis (MPGN); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) |
Apellis Pharmaceuticals, Inc. |
2023-05-29 | PHASE3 |
| NCT07215390 | RECRUITING | Geographic Atrophy Secondary to Age-related Macular Degeneration |
Apellis Pharmaceuticals, Inc. |
2025-06-23 | PHASE2 |
| NCT07495722 | NOT_YET_RECRUITING | Acute Ischemic Stroke; Cerebral Infarction |
Columbia University |
2026-10 | PHASE1 |
| NCT06722157 | ACTIVE_NOT_RECRUITING | Macular Degeneration, Age-related; Geographic Atrophy |
Boehringer Ingelheim |
2025-01-06 | PHASE2 |
| NCT07214740 | COMPLETED | Geographic Atrophy Secondary to Age-related Macular Degeneration |
Apellis Pharmaceuticals, Inc. |
2025-10-24 | PHASE3 |
| NCT04770545 | COMPLETED | Geographic Atrophy Secondary to Age-related Macular Degeneration |
Apellis Pharmaceuticals, Inc. |
2021-03-04 | PHASE3 |
| NCT03531255 | COMPLETED | PNH |
Apellis Pharmaceuticals, Inc. |
2018-08-27 | PHASE3 |
| NCT05148299 | COMPLETED | Transplant-Associated Thrombotic Microangiopathy |
Swedish Orphan Biovitrum |
2022-02-01 | PHASE2 |
| NCT05067127 | COMPLETED | C3G; IC-MPGN; C3 Glomerulopathy; C3 Glomerulonephritis; Complement 3 Glomerulopathy; Complement 3 Glomerulopathy (C3G); Complement 3 Glomerulonephritis; Dense Deposit Disease; DDD; Membranoproliferative Glomerulonephritis; Membranoproliferative Glomerulonephritis (MPGN); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) |
Apellis Pharmaceuticals, Inc. |
2021-11-12 | PHASE3 |
| NCT05096403 | COMPLETED | Cold Agglutinin Disease |
Swedish Orphan Biovitrum |
2022-10-20 | PHASE3 |
| NCT04579666 | TERMINATED | Amyotrophic Lateral Sclerosis; Motor Neuron Disease |
Apellis Pharmaceuticals, Inc. |
2020-09-30 | PHASE2 |
| NCT04572854 | COMPLETED | C3G; IC-MPGN; Renal Transplant; Complement 3 Glomerulopathy; Complement 3 Glomerulopathy (C3G); Dense Deposit Disease (DDD); Membranoproliferative Glomerulonephritis; Membranoproliferative Glomerulonephritis (MPGN); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN); C3 Glomerulopathy; C3 Glomerulonephritis; Complement 3 Glomerulonephritis |
Apellis Pharmaceuticals, Inc. |
2021-09-07 | PHASE2 |
| NCT03525613 | COMPLETED | Geographic Atrophy |
Apellis Pharmaceuticals, Inc. |
2018-08-31 | PHASE3 |
| NCT04085601 | COMPLETED | Paroxysmal Nocturnal Hemoglobinuria |
Apellis Pharmaceuticals, Inc. |
2019-08-27 | PHASE3 |
| NCT03525600 | COMPLETED | Geographic Atrophy |
Apellis Pharmaceuticals, Inc. |
2018-08-31 | PHASE3 |
| NCT03777332 | COMPLETED | Geographic Atrophy |
Apellis Pharmaceuticals, Inc. |
2018-11-05 | PHASE1 |
| NCT03500549 | COMPLETED | Paroxysmal Nocturnal Hemoglobinuria |
Apellis Pharmaceuticals, Inc. |
2018-06-14 | PHASE3 |
| NCT03593200 | COMPLETED | PNH |
Apellis Pharmaceuticals, Inc. |
2018-08-16 | PHASE2 |
| NCT02588833 | COMPLETED | Paroxysmal Nocturnal Hemoglobinuria |
Apellis Pharmaceuticals, Inc. |
2015-12-01 | PHASE1 |
| NCT03465709 | TERMINATED | Neovascular Age-related Macular Degeneration |
Apellis Pharmaceuticals, Inc. |
2018-02-14 | PHASE1; PHASE2 |
| NCT02264639 | COMPLETED | Paroxysmal Nocturnal Hemoglobinuria (PNH) |
Apellis Pharmaceuticals, Inc. |
2015-02-23 | PHASE1 |
| NCT02503332 | COMPLETED | Geographic Atrophy |
Apellis Pharmaceuticals, Inc. |
2015-09-24 | PHASE2 |
| NCT02461771 | COMPLETED | Neovascular Age-Related Macular Degeneration |
Apellis Pharmaceuticals, Inc. |
2015-01-28 | PHASE1 |
(data from https://clinicaltrials.gov, updated on 2026-08-05)
Check the
Pegcetacoplan acetate
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
Note: Technical data last updated: Sep 1, 2026.