paroxysmal nocturnal hemoglobinuria (PNH) | Page 8 | Aplastic Anemia & MDS International Foundation

paroxysmal nocturnal hemoglobinuria (PNH)

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not recently received complement inhibitor therapy.

The secondary objectives of the study are:

Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a multiple-center, open-label, multiple dose study to assess the efficacy, safety, and PK of the oral FD inhibitor ACH-0145228 in patients with PNH as monotherapy and on top of background therapy with an approved C5 inhibitor. After signing consent, subjects will return for periodic visits through Week 12, at which time the primary endpoint and key secondary assessments will be analyzed. Subjects will continue on treatment past 12 weeks into a long term extension portion of the trial.

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

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).

A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria

This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.