NCT05630001

Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Have Hb≥10 g/dL in Response to Anti-C5 Antibody and Switch to Iptacopan

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study was to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switched from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.

Detailed description

This was a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients. This study was comprised of two periods: * A Screening period lasting up to 8 weeks. * A 24-week open-label, iptacopan Treatment period. After completion of the treatment period, participants who continued to benefit from the iptacopan treatment based on the study doctor's evaluation were able to join the Roll-over extension study (CLNP023C12001B).

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	Iptacopan	Treatment with iptacopan at a dose of 200 mg b.i.d. will start on the first day (Day 1) and continue for 24 weeks.

Timeline

• Start date: 2023-04-24
• Primary completion: 2024-10-17
• Completion: 2024-10-17
• First posted: 2022-11-29
• Last updated: 2026-01-13
• Results posted: 2025-12-15

Locations

23 sites across 8 countries: United States, France, Germany, Italy, South Korea, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study