Iptacopan
Last Updated: 04/28/2026
An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age
Summary: The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \< 18 years of age.
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
Summary: This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes. The primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 month...
An Open-label, Non-randomized Extension Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Iptacopan (LNP023) in C3 Glomerulopathy or Idiopathic Immune-complex-membranoproliferative Glomerulonephritis
Summary: This is an open-label extension study to evaluate the long-term efficacy, safety and tolerability of iptacopan in subjects with C3 glomerulopathy or idiopathic immune-complex-membranoproliferative glomerulonephritis
Specified Drug-use Surveillance of Fabhalta Capsules (Paroxysmal Nocturnal Hemoglobinuria, CLNP023C11401)
Summary: This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.
Fabhalta Capsules Specified Drug-use Survey(C3 Glomerulopathy, CLNP023B11401)
Summary: The objective of this study is to evaluate the safety and effectiveness of Fabhalta in patients with C3 glomerulopathy in clinical practice.
A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Idiopathic Immune-complex-mediated Membranoproliferative Glomerulonephritis (IC-MPGN)
Summary: This study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis.
A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS
Summary: The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.
A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)
Summary: The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to \<18 years.
A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.
Summary: The Primary Completion Date and Study Completion Date have been updated to reflect completion of the adolescent cohort, which has been added to the protocol. The study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy.
A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase
Summary: The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC...
A Multicenter, Single Arm, Open Label Biopsy Study to Evaluate Structural and Functional Changes in Kidneys of Adult Patients With IgA Nephropathy Receiving Iptacopan on Top of Supportive Care
Summary: A study to investigate the impact of iptacopan treatment on the underlying immunopathology in patients with IgAN by assessing changes in key clinical and molecular markers from baseline to 9 months. The study aims to provide insights into the treatment's systemic and kidney-specific aspects by quantifying the change in mesangial C3c containing fragments deposition, as an indicator of complement ac...
An Adaptive, Randomized, Double-blind, Dose Exploration, Parallel Group, Placebo Controlled, Multicenter Phase 2 Trial to Evaluate the Efficacy, Safety and Tolerability of LNP023 in Combination With Standard-of-care With and Without Oral Corticosteroids in Patients With Active Lupus Nephritis Class III-IV, +/- V
Summary: The overall purpose of this two-part study is to evaluate the efficacy, safety and tolerability of iptacopan (LNP023) in addition to standard of care treatment.
Last Updated: 04/28/2026