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Clinical Trials in the USA / NCT06298552
Active, not recruiting Phase 3

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

NCT06298552 · tracked via the Priya Life Science USA tracker
Sponsor
argenx
Phase
Phase 3
Started
2024-04-16
Last updated
2026-08-10

Condition(s) studied

Generalized Myasthenia GravisgMGMyasthenia Gravis, GeneralizedMyasthenia Gravis

Investigational drug(s) / intervention(s)

Efgartigimod IVPlacebo IV

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)

Trial sites (91)

FacilityCityRegionStatus
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.

View NCT06298552 on ClinicalTrials.gov ↗ ← All trials in the USA