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

NCT ID: NCT03030183

Last Updated: 2022-07-27

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-04-17

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 RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.

Detailed Description

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Conditions

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Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Zilucoplan (RA101495)

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

Group Type EXPERIMENTAL

Zilucoplan (RA101495)

Intervention Type DRUG

0.3 mg/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)

0.3 mg/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
* 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ra Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Dr. Anita Hill

Role: STUDY_CHAIR

St James' Institute of Oncology

Locations

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Investigative Site

Duarte, California, United States

Site Status

Investigative Site

Los Angeles, California, United States

Site Status

Investigative Site

Chicago, Illinois, United States

Site Status

Investigative Site

Manhasset, New York, United States

Site Status

Investigative Site

Durham, North Carolina, United States

Site Status

Investigative Site

Dallas, Texas, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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RA101495-01.203

Identifier Type: -

Identifier Source: org_study_id

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