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

NCT ID: NCT03225287

Last Updated: 2023-09-28

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

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-17

Study Completion Date

2021-10-26

Brief Summary

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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.

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

Group Type EXPERIMENTAL

Zilucoplan (RA101495)

Intervention Type DRUG

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

Interventions

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Zilucoplan (RA101495)

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ra Pharmaceuticals, Inc.

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 4

Los Angeles, California, United States

Site Status

Investigative Site 19

Dallas, Texas, United States

Site Status

Investigative Site 3

Gosford, , Australia

Site Status

Investigative Site 5

Melbourne, , Australia

Site Status

Investigative Site 10

Toronto, , Canada

Site Status

Investigative Site 14

Helsinki, , Finland

Site Status

Investigative Site 9

Ulm, , Germany

Site Status

Investigative Site 17

Budapest, , Hungary

Site Status

Investigative Site 13

Christchurch, , New Zealand

Site Status

Investigative Site 12

Hamilton, , New Zealand

Site Status

Investigative Site 6

Leeds, , United Kingdom

Site Status

Investigative Site 7

London, , United Kingdom

Site Status

Countries

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United States Australia Canada Finland Germany Hungary New Zealand United Kingdom

References

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Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S, Griffin M, Korper S, Mikala G, Muus P, Overgaard U, Patriquin CJ, Pullon H, Shen YM, Spearing R, Szer J, De la Borderie G, Duda PW, Farzaneh-Far R, Ragunathan S, Sayegh CE, Vadysirisack DD, Schrezenmeier H. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. Haematologica. 2024 Mar 1;109(3):929-935. doi: 10.3324/haematol.2022.281780. No abstract available.

Reference Type RESULT
PMID: 37534517 (View on PubMed)

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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2016-003523-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RA101495-01.202

Identifier Type: -

Identifier Source: org_study_id

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