NCT04817618

Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 98 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The purpose of this study is to evaluate the efficacy and safety of iptacopan compared to placebo and standard of care in patients with native C3G. CLNP023B12301 is a Phase 3 pivotal trial for registration of iptacopan in C3G. The study aims to determine the reduction in UPCR and improvement in eGFR in participants treated with iptacopan compared to placebo, as well as the proportion of participants who achieve a composite renal endpoint consisting of eGFR and UPCR elements. These effects of iptacopan in conjunction with increases in serum C3 levels will provide support for an iptacopan profile that includes stabilization of eGFR, clinically meaningful reductions in proteinuria and inhibition of the complement AP. Kidney biopsies will be performed in adult participants to evaluate histopathological improvements in immunofluorescence and light microscopy that support these functional benefits of iptacopan.

Conditions

• C3G

Interventions

Type	Name	Description
DRUG	Placebo	Placebo to iptacopan 200mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)
DRUG	iptacopan	iptacopan 200 mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Timeline

• Start date: 2021-07-28
• Primary completion: 2027-01-29
• Completion: 2027-01-29
• First posted: 2021-03-26
• Last updated: 2026-04-01

Locations

87 sites across 19 countries: United States, Argentina, Belgium, Brazil, Canada, China, Czechia, France, Germany, Greece, India, Israel, Italy, Japan, Netherlands, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study