Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 3 evaluate LNP023 (iptacopan) across complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy, IgA nephropathy, age-related macular degeneration, ANCA-associated vasculitis, and hepatic impairment. Sponsored primarily by Novartis Pharmaceuticals alongside academic institutions, these protocols encompass various recruitment statuses ranging from recruiting and completed to active not recruiting and terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03955445 RECRUITING
C3 Glomerulopathy; Immune-complex-membranoproliferative Glomerulonephritis
Novartis Pharmaceuticals
 2019-10-03 PHASE3
NCT05795140 RECRUITING
Atypical Hemolytic Uremic Syndrome
Novartis Pharmaceuticals
 2024-05-08 PHASE3
NCT06934967 RECRUITING
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis Pharmaceuticals
 2025-10-28 PHASE3
NCT04557462 ACTIVE_NOT_RECRUITING
Primary IgA Nephropathy
Novartis Pharmaceuticals
 2021-09-20 PHASE3
NCT06994845 RECRUITING
Primary Immunoglobulin A Nephropathy (IgAN)
Novartis Pharmaceuticals
 2025-11-27 PHASE3
NCT05755386 RECRUITING
IC-MPGN
Novartis Pharmaceuticals
 2023-10-02 PHASE3
NCT05935215 RECRUITING
Atypical Hemolytic Uremic Syndrome
Novartis Pharmaceuticals
 2024-02-28 PHASE3
NCT07347990 RECRUITING
Thrombotic Microangiopathy; Hematopoietic Stem Cell Transplantation (HSCT)
First Affiliated Hospital of Zhejiang University
 2026-08-25
NCT06797518 RECRUITING
Glomerulonephritis, IGA; Immunoglobulin A Nephropathy
Novartis Pharmaceuticals
 2025-02-21 PHASE2
NCT04747613 ACTIVE_NOT_RECRUITING
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals
 2021-07-27 PHASE3
NCT06517758 ACTIVE_NOT_RECRUITING
Generalized Myasthenia Gravis
Novartis Pharmaceuticals
 2024-07-31 PHASE3
NCT04817618 RECRUITING
C3G
Novartis Pharmaceuticals
 2021-07-28 PHASE3
NCT06388941 ACTIVE_NOT_RECRUITING
Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis
Novartis Pharmaceuticals
 2024-08-05 PHASE2
NCT05230537 ACTIVE_NOT_RECRUITING
Age-Related Macular Degeneration
Novartis Pharmaceuticals
 2022-02-17 PHASE2
NCT04889430 COMPLETED
Atypical Hemolytic Uremic Syndrome
Novartis Pharmaceuticals
 2022-01-17 PHASE3
NCT05268289 RECRUITING
Lupus Nephritis
Novartis Pharmaceuticals
 2022-08-10 PHASE2
NCT06847607 NOT_YET_RECRUITING
AIHA - Warm Autoimmune Hemolytic Anemia; AIHA - Cold Autoimmune Hemolytic Anemia
Bing Han
 2025-03-01
NCT04578834 COMPLETED
IgA Nephropathy
Novartis Pharmaceuticals
 2021-01-25 PHASE3
NCT05630001 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals
 2023-04-24 PHASE3
NCT05086744 TERMINATED
Immune Thrombocytopenia (ITP); Cold Agglutinin Disease (CAD)
Novartis Pharmaceuticals
 2021-12-21 PHASE2
NCT05630001 Recruiting
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals|Novartis
 2023-04-24 Phase 3
NCT04154787 TERMINATED
Idiopathic Membranous Nephropathy
Novartis Pharmaceuticals
 2019-11-23 PHASE2
NCT04820530 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis Pharmaceuticals
 2021-07-19 PHASE3
NCT04558918 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis Pharmaceuticals
 2021-01-25 PHASE3
NCT05078580 COMPLETED
Hepatic Impairment
Novartis Pharmaceuticals
 2021-11-10 PHASE1
NCT02534909 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals
 2015-09-09 PHASE2
NCT05230537 Recruiting
Age-Related Macular Degeneration
Novartis Pharmaceuticals|Novartis
 2022-02-17 Phase 2
NCT05078580 Completed
Hepatic Impairment
Novartis Pharmaceuticals|Novartis
 2021-11-10 Phase 1
NCT03832114 COMPLETED
Glomerulonephritis
Novartis Pharmaceuticals
 2019-02-20 PHASE2
NCT04558918 Completed
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis Pharmaceuticals|Novartis
 2021-01-25 Phase 3
NCT03373461 COMPLETED
IgA Nephropathy
Novartis Pharmaceuticals
 2018-02-07 PHASE2
NCT03439839 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
Novartis Pharmaceuticals
 2018-04-09 PHASE2
NCT03896152 COMPLETED
Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals
 2019-04-05 PHASE2
NCT03955445 Recruiting
C3 Glomerulopathy
Novartis Pharmaceuticals|Novartis
 2019-10-03 Phase 3
NCT03832114 Completed
Glomerulonephritis
Novartis Pharmaceuticals|Novartis
 2019-02-20 Phase 2

(data from https://clinicaltrials.gov, updated on 2026-08-07)

Check the LNP023 (Iptacopan) Hydrochloride product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

LNP023 (Iptacopan) selectively binds complement factor B, inhibiting its catalytic activity and blocking the assembly of the alternative pathway C3 convertase. This targeted inhibition suppresses the alternative complement cascade amplifier loop, thereby preventing complement-mediated intravascular and extravascular hemolysis as well as renal tissue damage in conditions such as paroxysmal nocturnal hemoglobinuria, IgA nephropathy, and C3 glomerulopathy.

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>).

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Note: Technical data last updated: Sep 1, 2026.