Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

NCT ID: NCT03315130

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

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-11

Study Completion Date

2020-11-19

Brief Summary

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The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.

Detailed Description

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Conditions

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Generalized Myasthenia Gravis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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0.1 mg/kg zilucoplan (RA101495)

Group Type EXPERIMENTAL

zilucoplan (RA101495)

Intervention Type DRUG

Daily subcutaneous (SC) injection

0.3 mg/kg zilucoplan (RA101495)

Group Type EXPERIMENTAL

zilucoplan (RA101495)

Intervention Type DRUG

Daily subcutaneous (SC) injection

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Daily subcutaneous (SC) injection

Interventions

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zilucoplan (RA101495)

Daily subcutaneous (SC) injection

Intervention Type DRUG

Placebo

Daily subcutaneous (SC) injection

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of gMG \[Myasthenia Gravis Foundation of America (MGFA) Class II-IVa\] at Screening
* Positive serology for acetylcholine receptor (AChR) autoantibodies
* QMG score ≥ 12 at Screening and Randomization
* No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
* No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period

Exclusion Criteria

* Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
* History of meningococcal disease
* Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 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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UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273 (UCB)

Locations

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Diagnostic and Medical Clinic - Mobile

Mobile, Alabama, United States

Site Status

The Research Center of Southern California

Carlsbad, California, United States

Site Status

UCLA Medical Center

Los Angeles, California, United States

Site Status

University of California Irvine Health ALS and Neuromuscular Center

Orange, California, United States

Site Status

Yale University

New Haven, Connecticut, United States

Site Status

George Washington University

Washington D.C., District of Columbia, United States

Site Status

University of Florida

Jacksonville, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

University of Maryland

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Lahey Hospital and Medical Center

Burlington, Massachusetts, United States

Site Status

Wayne State University

Detroit, Michigan, United States

Site Status

Michigan State University

East Lansing, Michigan, United States

Site Status

University of Buffalo

Buffalo, New York, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

Mount Sinai Hospital

New York, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Allegheny Neurological Associates

Pittsburgh, Pennsylvania, United States

Site Status

Wesley Neurology Clinic

Cordova, Tennessee, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Vermont

Burlington, Vermont, United States

Site Status

Center for Neurological Disorders

Milwaukee, Wisconsin, United States

Site Status

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status

London Health Sciences Centre University Hospital

London, Ontario, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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Howard JF Jr, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ; Zilucoplan MG Study Group; Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar MG, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib AA, Mathew V, Mozaffar T, Hinton JL, Hewitt W, Barnett D, Sullivan P, Ho D, Howard JF Jr, Traub RE, Chopra M, Kaminski HJ, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon LM, Lisak RP, Bernitsas E, Jia K, Malik R, Lewis-Collins TD, Nicolle M, Nowak RJ, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe GI, Silvestri N, Patrick K, Zakalik K, Duda PW, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):582-592. doi: 10.1001/jamaneurol.2019.5125.

Reference Type RESULT
PMID: 32065623 (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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RA101495-02.201

Identifier Type: -

Identifier Source: org_study_id

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