Last updated on August 2019

Study of Safety Efficacy Tolerability Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)


Brief description of study

The main purpose of this study is to evaluate the efficacy of LNP023 in patients with PNH, showing signs of active hemolysis despite treatment with SoC (defined as an antibody with anti C5 activity).

Detailed Study Description

LNP023 is a novel oral small molecular weight compound, that inhibits alternative complement pathway (AP). Blockade of the AP with oral LNP023 has the potential to prevent both intra - and extravascular hemolysis.

This study includes a screening period of up to 68 days, a baseline visit, up to approximately 3 years of treatment with LNP023 administered in addition to SoC, and an End of Study (EoS) visit 2 weeks after last LNP023 administration.

The main purpose of this study is to evaluate the efficacy of LNP023 in patients with PNH, showing signs of active hemolysis despite treatment with SoC (defined as an antibody with anti C5 activity).

Clinical Study Identifier: NCT03439839

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