NCT05935215

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 14 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period. The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.

Conditions

• Atypical Hemolytic Uremic Syndrome

Interventions

Type	Name	Description
DRUG	Iptacopan	Open Label

Timeline

• Start date: 2024-02-28
• Primary completion: 2028-07-21
• Completion: 2029-07-19
• First posted: 2023-07-07
• Last updated: 2026-04-01

Locations

31 sites across 8 countries: China, France, Germany, Italy, Japan, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study