Title
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab
Phase
Phase 2Lead Sponsor
Ra PharmaceuticalsStudy Type
InterventionalStatus
Completed Results PostedIndication/Condition
Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention/Treatment
zilucoplan ...Study Participants
3The 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.
0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Subjects will receive RA101495 at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
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
| Event Type | Organ System | Event Term | Zilucoplan (RA101495) |
|---|
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Changes from baseline at each of the scheduled postbaseline time-points
Changes from baseline at each of the scheduled postbaseline time-points
Changes from baseline at each of the scheduled postbaseline time-points
Changes from baseline at each of the scheduled postbaseline time-points
Changes from baseline at each of the scheduled postbaseline time-points Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10. Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
Changes from baseline at each of the scheduled postbaseline time-points
