At a glance
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An Open Label, Single Arm, Multiple Dose Study to Assess Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of LNP023 When Administered in Addition to Standard of Care (SoC) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
In Brief
A Phase 2 clinical trial evaluating iptacopan and Standard of Care for Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis. Completed, enrolled 16 participants across 3 sites in 3 countries.
Detailed Summary
This was a Phase 2, open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of iptacopan when administered in addition to Standard of care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.
Study Details
Timeline
Interventions
iptacopan bid orally administered
Standard of Care (SoC) is defined as an antibody with anti C5 activity. At the time of study start, eculizumab was the only available SoC; eculizumab will be hereafter referred to as SoC.