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

NCT ID: NCT06298552

Last Updated: 2025-11-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

119 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-16

Study Completion Date

2027-06-30

Brief Summary

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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) 2.5 years.

Detailed Description

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Conditions

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Generalized Myasthenia Gravis gMG Myasthenia Gravis, Generalized Myasthenia Gravis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Efgartigimod IV

Patients receiving efgartigimod IV in both part A and part B

Group Type EXPERIMENTAL

Efgartigimod IV

Intervention Type BIOLOGICAL

Intravenous infusion of efgartigimod

Placebo

Patients receiving placebo during part A and receiving efgartigimod IV during part B

Group Type PLACEBO_COMPARATOR

Efgartigimod IV

Intervention Type BIOLOGICAL

Intravenous infusion of efgartigimod

Placebo IV

Intervention Type OTHER

Intravenous infusion of placebo

Interventions

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Efgartigimod IV

Intravenous infusion of efgartigimod

Intervention Type BIOLOGICAL

Placebo IV

Intravenous infusion of placebo

Intervention Type OTHER

Eligibility Criteria

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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 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 that includes 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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HonorHealth Neurology - Bob Bove Neuroscience Institute

Scottsdale, Arizona, United States

Site Status

Loma Linda University Health

Fresno, California, United States

Site Status

First Choice Neurology Boca Raton

Boca Raton, Florida, United States

Site Status

SFM Clinical Research LLC

Boca Raton, Florida, United States

Site Status

The Neurology Institute / Healthcare Innovations Institute - Coral Springs

Coral Springs, Florida, United States

Site Status

Neurology Associates PA

Maitland, Florida, United States

Site Status

Desai Sethi Medical Center

Miami, Florida, United States

Site Status

Medsol Clinical Research Center Inc

Port Charlotte, Florida, United States

Site Status

BayCare - St. Anthony's Hospital

St. Petersburg, Florida, United States

Site Status

University of South Florida (USF) Health - Morsani Center for Advanced Healthcare

Tampa, Florida, United States

Site Status

Wellstar - Augusta University Medical Center

Augusta, Georgia, United States

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Kansas University Medical Center - Kansas City

Fairway, Kansas, United States

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Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

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University of North Carolina - Chapel Hill

Chapel Hill, North Carolina, United States

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Duke University School of Medicine - Duke Early Phase Clinical Research Unit

Durham, North Carolina, United States

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Cleveland Clinic

Cleveland, Ohio, United States

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Erlanger Neuroscience Institute

Chattanooga, Tennessee, United States

Site Status

National Neuromuscular Research Institute

Austin, Texas, United States

Site Status

University of Washington Medical Center - Montlake

Seattle, Washington, United States

Site Status

Hôpital Erasme

Anderlecht, , Belgium

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Cliniques Universitaires Saint-Luc

Brussels, , Belgium

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AZ Sint-Lucas Gent

Ghent, , Belgium

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UZ Leuven

Leuven, , Belgium

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Heritage Medical Research Clinic

Calgary, , Canada

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Genge Partners Inc.

Montreal, , Canada

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Ottawa Hospital - Civic Campus

Ottawa, , Canada

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Xuanwu Hospital Capital Medical University

Beijing, , China

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The First Hospital of Jilin University

Changchun, , China

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Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital

Chongqing, , China

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The First Affiliated Hospital of Chongqing Medical University

Chongqing, , China

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Fujian Medical University Union Hospital

Fuzhou, , China

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The First Affiliated Hospital of Guangxi Medical University

Guangxi, , China

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The First Affiliated Hospital of Guangzhou University Chinese Medicine

Guangzhou, , China

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Guangdong Province Traditional Chinese Medical Hospital

Guangzhou, , China

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The Affiliated Hospital of Guizhou Medical University

Guiyang, , China

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Ningbo Medical Center Lihuili Hospital

Ningbo, , China

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Qilu Hospital of Shandong University

Qingdao, , China

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Huashan Hospital Fudan University

Shanghai, , China

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First Hospital of Shanxi Medical University

Taiyuan, , China

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Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, , China

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Cyprus Institute of Neurology and Genetics

Égkomi, , Cyprus

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Fakultni nemocnice Brno

Brno, , Czechia

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Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice

Pardubice, , Czechia

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Aarhus Universitetshospital

Aarhus, , Denmark

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Rigshospitalet

Copenhagen, , Denmark

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Odense Universitetshospital

Odense C, , Denmark

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Turun Yliopistollinen Keskussairaala

Turku, , Finland

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AP-HM- Hôpital de La Timone

Marseille, , France

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CHU de Nice-Hôpital Pasteur

Nice, , France

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AP-HP - Hôpital de la Pitié Salpétrière

Paris, , France

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West Georgia Medical Center

Kutaisi, , Georgia

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Petre Sarajishvili Institute of Neurology

Tbilisi, , Georgia

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Pineo Medical Ecosystem

Tbilisi, , Georgia

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New Hospitals

Tbilisi, , Georgia

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LLC Caucasus Medical Centre

Tbilisi, , Georgia

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Charité - Universitätsmedizin Berlin

Berlin, , Germany

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Katholisches Klinikum Bochum - St. Josef Hospital

Bochum, , Germany

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Universitätsklinikum Würzburg

Würzburg, , Germany

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Eginitio Hospital

Athens, , Greece

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University General Hospital ''ATTIKON''

Chaïdári, , Greece

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University General Hospital of Patras

Pátrai, , Greece

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AHEPA University General Hospital of Thessaloniki

Thessaloniki, , Greece

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Semmelweis Egyetem Genomikai Medicina és Ritka Betegségek Intézete

Budapest, , Hungary

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Leiden University Medical Center

Leiden, , Netherlands

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Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

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Haukeland Universitetssykehus

Bergen, , Norway

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Oslo Universitetssykehus HF, Ullevål

Oslo, , Norway

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MICS Centrum Medyczne Bydgoszcz

Bydgoszcz, , Poland

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Centrum Medyczne Neurologia Slaska

Katowice, , Poland

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Szpital Uniwersytecki w Krakowie

Krakow, , Poland

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Krakowska Akademia Neurologii Sp. z o.o.

Krakow, , Poland

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CLINIREM Sp z o.o.

Lublin, , Poland

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Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

Wroclaw, , Poland

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ULS de Santa Maria, EPE - Hospital de Santa Maria

Lisbon, , Portugal

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ULS de Lisboa Ocidental, EPE - Hospital Egas Moniz

Lisbon, , Portugal

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Hospital de Santo António - Unidade Local de Saúde de Santo António

Porto, , Portugal

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Cluj County Emergency Clinical Hospital

Cluj-Napoca, , Romania

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Sibiu Emergency County Clinical Hospital

Sibiu, , Romania

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King Faisal Specialist Hospital & Research Center

Riyadh, , Saudi Arabia

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National Guard Riyadh

Riyadh, , Saudi Arabia

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Opsta bolnica MSB - Medicinski Sistemi Beograd

Belgrade, , Serbia

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University Clinical Center Kragujevac

Kragujevac, , Serbia

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Hospital General Universitario Dr. Balmis

Alicante, , Spain

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Hospital Universitario Vall d'Hebron - PPDS

Barcelona, , Spain

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Hospital de La Santa Creu i Sant Pau

Barcelona, , Spain

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Hospital Universitari Arnau de Vilanova

Lleida, , Spain

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Hospital Universitario Ramon y Cajal

Madrid, , Spain

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Hospital Clinico Universitario de Santiago

Santiago de Compostela, , Spain

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Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

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Panthera Biopartners

Glasgow, , United Kingdom

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Leeds General Infirmary

Leeds, , United Kingdom

Site Status

Countries

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United States Belgium Canada China Cyprus Czechia Denmark Finland France Georgia Germany Greece Hungary Netherlands Norway Poland Portugal Romania Saudi Arabia Serbia Spain United Kingdom

Related Links

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Other Identifiers

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2024-511796-15-00

Identifier Type: OTHER

Identifier Source: secondary_id

ARGX-113-2308

Identifier Type: -

Identifier Source: org_study_id

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