Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients

Ra Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Treatments: Drug: Zilucoplan (RA101495)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03078582

2016-003522-16 (EudraCT Number)

RA101495-01.201

Details and patient eligibility

About

The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.

Enrollment: 26 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of PNH by flow cytometry
• For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level ≥2 times the upper limit of normal (xULN) during Screening
• For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening

Exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

26 participants in 2 patient groups

Zilucoplan (RA101495) treatment naive

Experimental group

Description:

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

Treatment:

Drug: Zilucoplan (RA101495)

Zilucoplan (RA101495) previously on eculizumab

Experimental group

Description:

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

Treatment:

Drug: Zilucoplan (RA101495)