Multiple clinical trials evaluate the therapeutic profile, safety, and pharmacokinetics of avacopan (CCX168) across Phase 1 through Phase 4 studies targeting conditions such as anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, including granulomatosis with polyangiitis, end-stage renal disease, and hepatic impairment. Led by key industry sponsors—including Amgen, Vifor Fresenius Medical Care Renal Pharma, and Kissei Pharmaceutical—alongside academic centers such as the Mayo Clinic and Toulouse University Hospital, these initiatives range from completed pharmacokinetic evaluations in healthy volunteers to recruiting and upcoming Phase 3 and Phase 4 trials in patients with severe renal involvement or systemic vasculitis.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06072482 | RECRUITING | Antineutrophil Cytoplasmic Antibody-associated Vasculitis |
Amgen |
2024-02-07 | PHASE4 |
| NCT07176546 | RECRUITING | Granulomatosis With Polyangiitis; Wegener's; GPA |
Robert Spiera, MD |
2026-05-22 | PHASE2; PHASE3 |
| NCT07373262 | NOT_YET_RECRUITING | ANCA-Associated Vasculitis (AAV) |
University Hospital, Toulouse |
2026-03-01 | PHASE3 |
| NCT06676579 | RECRUITING | IgA Nephropathy (IgAN) |
Mayo Clinic |
2025-06-09 | PHASE2 |
| NCT07556484 | RECRUITING | Diffuse Alveolar Hemorrhage; Antineutrophil Cytoplasmic Antibody Positive Vasculitis |
Mayo Clinic |
2026-08-01 | PHASE1 |
| NCT06321601 | ACTIVE_NOT_RECRUITING | Vasculitis |
Amgen |
2024-10-22 | PHASE3 |
| NCT06611696 | RECRUITING | ANCA Associated Vasculitis (AAV) |
Chiba University |
2024-11-15 | PHASE4 |
| NCT06468826 | COMPLETED | End-Stage Renal Disease (ESRD) |
Amgen |
2024-06-18 | PHASE1 |
| NCT06321601 | Not yet recruiting | Vasculitis |
Amgen |
2024-07-31 | Phase 3 |
| NCT05897684 | Recruiting | ANCA-associated Vasculitis |
Vifor Fresenius Medical Care Renal Pharma |
2023-09-11 | -- |
| NCT06207682 | COMPLETED | Healthy Volunteers |
Amgen |
2022-06-28 | PHASE1 |
| NCT06207682 | Completed | Healthy Volunteers |
Amgen |
2022-06-28 | Phase 1 |
| NCT03301467 | COMPLETED | C3 Glomerulopathy (C3G) |
Amgen |
2017-09-29 | PHASE2 |
| NCT03852472 | COMPLETED | Hidradenitis Suppurativa; Acne Inversa |
Amgen |
2018-12-21 | PHASE2 |
| NCT05988034 | COMPLETED | Electrocardiography |
Amgen |
2019-11-08 | PHASE1 |
| NCT02994927 | COMPLETED | ANCA-Associated Vasculitis |
Amgen |
2017-03-15 | PHASE3 |
| NCT06004934 | COMPLETED | Hepatic Impairment |
Amgen |
2018-04-17 | PHASE1 |
| NCT06004934 | Completed | Hepatic Impairment |
Amgen |
2018-04-17 | Phase 1 |
| NCT05988008 | Completed | Anti-neutrophil Cytoplasmic Antibody-associated Vasculitis |
Amgen |
2017-10-23 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2026-08-14)
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>).