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

Completed · Phase 2

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH)

In brief

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.

Key facts

Study ID

NCT03030183

Run by

Ra Pharmaceuticals

People needed

3

Starts

2017-04-17

Expected to finish

2018-03-28

Last updated by the study team

2022-07-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• History of meningococcal disease
• Current systemic infection or suspicion of active bacterial infection

Where it is running

• 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
• Investigative Site — Durham, North Carolina, United States
• Investigative Site — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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