NCT05795140

Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 125 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.

Detailed description

The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.

Conditions

• Atypical Hemolytic Uremic Syndrome

Interventions

Type	Name	Description
DRUG	Iptcaopan 200 mg	Open label, participant specific kits, hard gelatin capsules to be taken twice a day

Timeline

• Start date: 2024-05-08
• Primary completion: 2032-12-23
• Completion: 2033-01-03
• First posted: 2023-04-03
• Last updated: 2025-07-16

Locations

6 sites across 4 countries: Brazil, China, Czechia, India

Regulatory

• FDA-regulated drug study