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

Ra Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Treatments: Drug: Zilucoplan (RA101495)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03030183

RA101495-01.203

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

3 participants in 1 patient group

Zilucoplan (RA101495)

Experimental group

Description:

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

Treatment:

Drug: Zilucoplan (RA101495)