Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study

Ra Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Treatments: Drug: Zilucoplan (RA101495)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03225287

RA101495-01.202

2016-003523-34 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.

Enrollment: 19 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
• Evidence of ongoing clinical benefit in the opinion of the Investigator

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)

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19 participants in 1 patient group

Zilucoplan (RA101495)

Experimental group

Description:

Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study

Treatment:

Drug: Zilucoplan (RA101495)