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

• Status: Completed
• Trial end date: 2018-03-28
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ra Pharmaceuticals

Criteria

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