Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
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
The purpose of the study is to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.
This is a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients. This study is 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 continue to benefit from the iptacopan treatment based on the study doctor's evaluation will be able to join the Roll-over extension study. ;
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