NCT04747613

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 208 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

Detailed description

The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	Iptacopan	Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral

Timeline

• Start date: 2021-07-27
• Primary completion: 2027-10-18
• Completion: 2027-10-18
• First posted: 2021-02-10
• Last updated: 2026-04-16

Locations

60 sites across 17 countries: United States, Brazil, China, Czechia, France, Germany, Italy, Japan, Lithuania, Malaysia, Netherlands, Singapore, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study