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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

26 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-08

Study Completion Date

2018-03-28

Brief Summary

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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.

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Detailed Description

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Conditions

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Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Zilucoplan (RA101495) treatment naive

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)

Group Type EXPERIMENTAL

Zilucoplan (RA101495)

Intervention Type DRUG

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

Zilucoplan (RA101495) previously on eculizumab

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)

Group Type EXPERIMENTAL

Zilucoplan (RA101495)

Intervention Type DRUG

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

Interventions

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Zilucoplan (RA101495)

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

Intervention Type DRUG

Eligibility Criteria

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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