NCT03955445

Long-term Efficacy, Safety and Tolerability of Iptacopan in C3G or IC-MPGN

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 225 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 100 Years
• Healthy volunteers: Not accepted

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

Detailed description

The primary purpose of this extension study is to collect long-term efficacy, safety and tolerability data in eligible participants receiving open-label iptacopan after completing treatment in the C3G Phase 2 proof of concept study CLNP023X2202. The primary (at 9 months) and longer-term (\>9 months) efficacy and safety data of iptacopan collected from CLNP023X2202 participants will be used to support health authority submissions. This umbrella protocol will also allow: * continued access to iptacopan to patients enrolled in the ongoing Phase 3 programs (C3G and IC-MPGN) * C3G study (CLNP023B12301): adults and adolescents * IC-MPGN study (CLNP023B12302): adults and adolescents * provision of additional efficacy and safety information following longer-term treatment in C3G and IC-MPGN populations to support health authority submissions. Efficacy and safety assessments at the 9 month visit of this extension study in combination with data from CLNP023X2202 (baseline plus 3 months of treatment) allowed evaluation of the effects of iptacopan on potential endpoint(s) at 12 months of iptacopan treatment in C3G participants. The enrollment of C3G and IC-MPGN participants (adults and adolescents) from Phase 3 studies, CLNP023B12301 and CLNP023B12302, permits longer-term evaluation of the persistence of effects observed after iptacopan treatment. These longer term efficacy and safety assessments may be compared to historical/concurrent control data available from relevant real world databases in C3G or IC-MPGN patients and used as supportive information for registration purposes. This extension study is expected to continue until the drug product becomes commercially available and accessible (anticipated to be up to approximately 168 months from the first patient first visit date), or the benefit-risk profile is no longer positive, or the program is discontinued for business or strategic reasons. "Baseline" refers to the Day 1 visit (pre-dose) of CLNP023X2202, CLNP023B12301 or CLNP023B12302, whereas the Day 1 visit for this C3G/IC-MPGN extension study (CLNP023B12001B) is identified as "Extension Day 1".

Conditions

• C3 Glomerulopathy
• Immune-complex-membranoproliferative Glomerulonephritis

Interventions

Type	Name	Description
DRUG	LNP023	LNP023 capsules

Timeline

• Start date: 2019-10-03
• Primary completion: 2036-05-30
• Completion: 2036-05-30
• First posted: 2019-05-20
• Last updated: 2026-04-14

Locations

50 sites across 17 countries: United States, Argentina, Brazil, Canada, China, Czechia, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study