A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
Efgartigimod IV: Intravenous infusion of efgartigimod
Placebo IV: Intravenous infusion of placebo
Study summary
The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 3.5 years.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* The participant is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF.
* The participant is capable of providing signed informed consent and following with protocol requirements.
* The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the study drug.
* The participant has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG of both of the following:
1. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation (RNS) or is for anti-muscle-specific kinase antibodies (MuSK-Ab) seropositive
2. Either a history of positive edrophonium chloride test OR a demonstrated improvement in MG signs with treatments such as oral acetylcholinesterase (AChE) inhibitors, plasma exchange (PLEX), immunoabsorption, or intravenous immunoglobulin (IVIg)/ subcutaneous immunoglobulin (SCIg) treatment
* The participant is receiving a stable dose of MG therapy before screening, including acetylcholinesterase (AChE) inhibitors, steroids, or nonsteroidal immunosuppressive therapies (NSISTs) in combination or alone
Exclusion Criteria:
* Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or puts the participant at undue risk
* History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
* Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
* History of or current alcohol, drug, or medication abuse as assessed by the investigator
* Pregnant or lactating state or intention to become pregnant during the study
* Live or live-attenuated vaccine received \<4 weeks before screening
* Worsening muscle weakness secondary to concurrent infections or medications
* Received a thymectomy less than 3 months before screening or thymectomy is planned during the study
The complete list of exclusion criteria can be found in the protocol.
Primary outcome measure(s)
MG-ADL Total Score Change From Baseline — Up to 29 days in part A The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (no symptoms) to 24 (most severe symptoms).
Trial sites (91)
Facility
City
Region
Status
HonorHealth Neurology - Bob Bove Neuroscience Institute
Scottsdale
Arizona
Loma Linda University Health
Fresno
California
First Choice Neurology Boca Raton
Boca Raton
Florida
SFM Clinical Research LLC
Boca Raton
Florida
The Neurology Institute / Healthcare Innovations Institute - Coral Springs
Coral Springs
Florida
Neurology Associates PA
Maitland
Florida
Desai Sethi Medical Center
Miami
Florida
Medsol Clinical Research Center Inc
Port Charlotte
Florida
BayCare - St. Anthony's Hospital
St. Petersburg
Florida
University of South Florida (USF) Health - Morsani Center for Advanced Healthcare
Tampa
Florida
Wellstar - Augusta University Medical Center
Augusta
Georgia
Kansas University Medical Center - Kansas City
Fairway
Kansas
Rutgers-Robert Wood Johnson Medical School
New Brunswick
New Jersey
University of North Carolina - Chapel Hill
Chapel Hill
North Carolina
Duke University School of Medicine - Duke Early Phase Clinical Research Unit
Durham
North Carolina
Cleveland Clinic
Cleveland
Ohio
Erlanger Neuroscience Institute
Chattanooga
Tennessee
National Neuromuscular Research Institute
Austin
Texas
University of Washington Medical Center - Montlake
Seattle
Washington
Hôpital Erasme
Anderlecht
Belgium
Cliniques Universitaires Saint-Luc
Brussels
Belgium
AZ Sint-Lucas Gent
Ghent
Belgium
UZ Leuven
Leuven
Belgium
Heritage Medical Research Clinic
Calgary
Canada
Genge Partners Inc.
Montreal
Canada
Ottawa Hospital - Civic Campus
Ottawa
Canada
Xuanwu Hospital Capital Medical University
Beijing
China
The First Hospital of Jilin University
Changchun
China
Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital
Chongqing
China
The First Affiliated Hospital of Chongqing Medical University
Chongqing
China
Fujian Medical University Union Hospital
Fuzhou
China
The First Affiliated Hospital of Guangxi Medical University
Guangxi
China
The First Affiliated Hospital of Guangzhou University Chinese Medicine
Guangzhou
China
Guangdong Province Traditional Chinese Medical Hospital
Guangzhou
China
The Affiliated Hospital of Guizhou Medical University
Guiyang
China
Ningbo Medical Center Lihuili Hospital
Ningbo
China
Qilu Hospital of Shandong University
Qingdao
China
Huashan Hospital Fudan University
Shanghai
China
First Hospital of Shanxi Medical University
Taiyuan
China
Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan
China
+ 51 more sites — see the full list on the official registry below.
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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