Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis
1 other identifier
interventional
45
2 countries
30
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Oct 2017
Typical duration for phase_2
30 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 11, 2017
CompletedFirst Submitted
Initial submission to the registry
October 16, 2017
CompletedFirst Posted
Study publicly available on registry
October 19, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 10, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
November 19, 2020
CompletedResults Posted
Study results publicly available
June 27, 2022
CompletedJuly 27, 2022
July 1, 2022
1.2 years
October 16, 2017
May 31, 2022
July 26, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
From Baseline to Week 12
Secondary Outcomes (12)
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale
From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey
From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score
From Baseline to Week 12
Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12
Week 12
Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period
Up to Week 12
- +7 more secondary outcomes
Study Arms (3)
0.1 mg/kg zilucoplan (RA101495)
EXPERIMENTAL0.3 mg/kg zilucoplan (RA101495)
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
Daily subcutaneous (SC) injection
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (30)
Diagnostic and Medical Clinic - Mobile
Mobile, Alabama, 36604, United States
The Research Center of Southern California
Carlsbad, California, 92011, United States
UCLA Medical Center
Los Angeles, California, 90095, United States
University of California Irvine Health ALS and Neuromuscular Center
Orange, California, 92868, United States
Yale University
New Haven, Connecticut, 06520, United States
George Washington University
Washington D.C., District of Columbia, 20052, United States
University of Florida
Jacksonville, Florida, 32209, United States
University of South Florida
Tampa, Florida, 33620, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Maryland
Baltimore, Maryland, 21201, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Lahey Hospital and Medical Center
Burlington, Massachusetts, 01805, United States
Wayne State University
Detroit, Michigan, 48202, United States
Michigan State University
East Lansing, Michigan, 48864, United States
University of Buffalo
Buffalo, New York, 14260, United States
Hospital for Special Surgery
New York, New York, 10021, United States
Mount Sinai Hospital
New York, New York, 10029, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Ohio State University
Columbus, Ohio, 43210, United States
Allegheny Neurological Associates
Pittsburgh, Pennsylvania, 15212, United States
Wesley Neurology Clinic
Cordova, Tennessee, 38018, United States
University of Texas Southwestern
Dallas, Texas, 75390, United States
University of Utah
Salt Lake City, Utah, 84132, United States
University of Vermont
Burlington, Vermont, 05405, United States
Center for Neurological Disorders
Milwaukee, Wisconsin, 53215, United States
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7, Canada
London Health Sciences Centre University Hospital
London, Ontario, N6A 5A5, Canada
Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4, Canada
Related Publications (1)
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.
PMID: 32065623RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- UCB
- Organization
- Cares
Study Officials
- STUDY DIRECTOR
UCB Cares
001 844 599 2273 (UCB)
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 16, 2017
First Posted
October 19, 2017
Study Start
October 11, 2017
Primary Completion
December 10, 2018
Study Completion
November 19, 2020
Last Updated
July 27, 2022
Results First Posted
June 27, 2022
Record last verified: 2022-07
Data Sharing
- IPD Sharing
- Will not share