Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

Ra Pharmaceuticals3 enrolled

Overview

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Zilucoplan (RA101495)DRUG

0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of PNH by flow cytometry * Inadequate response to eculizumab defined as having received eculizumab for at least 6 months plus a documented LDH level ≥ 1.5 x the upper limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab Exclusion Criteria: * History of meningococcal disease * Current systemic infection or suspicion of active bacterial infection

Locations (6)

Investigative Site

Duarte, California, United States

Investigative Site

Los Angeles, California, United States

Investigative Site

Chicago, Illinois, United States

Investigative Site

Manhasset, New York, United States

Outcomes

Primary Outcomes

Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.

Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels

Time frame: Through Week 12 of the study