Clinical trial NCT06298552: A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
Official title: A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-03-07
Enrollment: 119
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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) 2.5 years.


Eligibility criteria: 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 \&lt;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 outcomes:
measure: Myasthenia Gravis Activities of Daily Living (MG-ADL) total score change from baseline
timeFrame: Up to 29 days during part A
Secondary outcomes:
measure: Quantitative myasthenia gravis (QMG) total score change from baseline
timeFrame: Up to 29 days during part A
measure: Proportion of participants who are both MG-ADL and QMG responders
timeFrame: Up to 8 weeks during part A
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: HonorHealth Neurology - Bob Bove Neuroscience Institute
state: Arizona
city: Fresno
country: United States
facility: Loma Linda University Health
state: California
city: Boca Raton
country: United States
facility: First Choice Neurology Boca Raton
state: Florida
city: Boca Raton
country: United States
facility: SFM Clinical Research LLC
state: Florida
city: Coral Springs
country: United States
facility: The Neurology Institute / Healthcare Innovations Institute - Coral Springs
state: Florida
city: Maitland
country: United States
facility: Neurology Associates PA
state: Florida
city: Miami
country: United States
facility: Desai Sethi Medical Center
state: Florida
city: Port Charlotte
country: United States
facility: Medsol Clinical Research Center Inc
state: Florida
city: Saint Petersburg
country: United States
facility: BayCare - St. Anthony's Hospital
state: Florida
city: Tampa
country: United States
facility: University of South Florida (USF) Health - Morsani Center for Advanced Healthcare
state: Florida
city: Augusta
country: United States
facility: Wellstar - Augusta University Medical Center
state: Georgia
city: Fairway
country: United States
facility: Kansas University Medical Center - Kansas City
state: Kansas
city: New Brunswick
country: United States
facility: Rutgers-Robert Wood Johnson Medical School
state: New Jersey
city: Chapel Hill
country: United States
facility: University of North Carolina - Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke University School of Medicine - Duke Early Phase Clinical Research Unit
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Chattanooga
country: United States
facility: Erlanger Neuroscience Institute
state: Tennessee
city: Austin
country: United States
facility: National Neuromuscular Research Institute
state: Texas
city: Seattle
country: United States
facility: University of Washington Medical Center - Montlake
state: Washington
city: Anderlecht
country: Belgium
facility: Hôpital Erasme
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Gent
country: Belgium
facility: AZ Sint-Lucas Gent
city: Leuven
country: Belgium
facility: UZ Leuven
city: Calgary
country: Canada
facility: Heritage Medical Research Clinic
city: Montreal
country: Canada
facility: Genge Partners Inc.
city: Ottawa
country: Canada
facility: Ottawa Hospital - Civic Campus
city: Beijing
country: China
facility: Xuanwu Hospital Capital Medical University
city: Changchun
country: China
facility: The First Hospital of Jilin University
city: Chongqing
country: China
facility: Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital
city: Chongqing
country: China
facility: The First Affiliated Hospital of Chongqing Medical University
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
city: Guangxi
country: China
facility: The First Affiliated Hospital of Guangxi Medical University
city: Guangzhou
country: China
facility: The First Affiliated Hospital of Guangzhou University Chinese Medicine
city: Guangzhou
country: China
facility: Guangdong Province Traditional Chinese Medical Hospital
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
city: Ningbo
country: China
facility: Ningbo Medical Center Lihuili Hospital
city: Qingdao
country: China
facility: Qilu Hospital of Shandong University
city: Shanghai
country: China
facility: Huashan Hospital Fudan University
city: Taiyuan
country: China
facility: First Hospital of Shanxi Medical University
city: Wuhan
country: China
facility: Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
city: Egkomi
country: Cyprus
facility: Cyprus Institute of Neurology and Genetics
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno
city: Pardubice
country: Czechia
facility: Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice
city: Aarhus
country: Denmark
facility: Aarhus Universitetshospital
city: Copenhagen
country: Denmark
facility: Rigshospitalet
city: Odense C
country: Denmark
facility: Odense Universitetshospital
city: Turku
country: Finland
facility: Turun Yliopistollinen Keskussairaala
city: Marseille
country: France
facility: AP-HM- Hôpital de La Timone
city: Nice
country: France
facility: CHU de Nice-Hôpital Pasteur
city: Paris
country: France
facility: AP-HP - Hôpital de la Pitié Salpétrière
city: Kutaisi
country: Georgia
facility: West Georgia Medical Center
city: Tbilisi
country: Georgia
facility: Petre Sarajishvili Institute of Neurology
city: Tbilisi
country: Georgia
facility: Pineo Medical Ecosystem
city: Tbilisi
country: Georgia
facility: New Hospitals
city: Tbilisi
country: Georgia
facility: LLC Caucasus Medical Centre
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Bochum
country: Germany
facility: Katholisches Klinikum Bochum - St. Josef Hospital
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg
city: Athens
country: Greece
facility: Eginitio Hospital
city: Chaidari
country: Greece
facility: University General Hospital ''ATTIKON''
city: Patra
country: Greece
facility: University General Hospital of Patras
city: Thessaloniki
country: Greece
facility: AHEPA University General Hospital of Thessaloniki
city: Budapest
country: Hungary
facility: Semmelweis Egyetem Genomikai Medicina és Ritka Betegségek Intézete
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Bergen
country: Norway
facility: Haukeland Universitetssykehus
city: Oslo
country: Norway
facility: Oslo Universitetssykehus HF, Ullevål
city: Bydgoszcz
country: Poland
facility: MICS Centrum Medyczne Bydgoszcz
city: Katowice
country: Poland
facility: Centrum Medyczne Neurologia Slaska
city: Kraków
country: Poland
facility: Szpital Uniwersytecki w Krakowie
city: Kraków
country: Poland
facility: Krakowska Akademia Neurologii Sp. z o.o.
city: Lublin
country: Poland
facility: CLINIREM Sp z o.o.
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
city: Lisboa
country: Portugal
facility: ULS de Santa Maria, EPE - Hospital de Santa Maria
city: Lisboa
country: Portugal
facility: ULS de Lisboa Ocidental, EPE - Hospital Egas Moniz
city: Porto
country: Portugal
facility: Hospital de Santo António - Unidade Local de Saúde de Santo António
city: Cluj Napoca
country: Romania
facility: Cluj County Emergency Clinical Hospital
city: Sibiu
country: Romania
facility: Sibiu Emergency County Clinical Hospital
city: Riyadh
country: Saudi Arabia
facility: King Faisal Specialist Hospital & Research Center
city: Riyadh
country: Saudi Arabia
facility: National Guard Riyadh
city: Belgrade
country: Serbia
facility: Opsta bolnica MSB - Medicinski Sistemi Beograd
city: Kragujevac
country: Serbia
facility: University Clinical Center Kragujevac
city: Alicante
country: Spain
facility: Hospital General Universitario Dr. Balmis
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron - PPDS
city: Barcelona
country: Spain
facility: Hospital de La Santa Creu i Sant Pau
city: Lleida
country: Spain
facility: Hospital Universitari Arnau de Vilanova
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Santiago de Compostela
country: Spain
facility: Hospital Clinico Universitario de Santiago
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
city: Glasgow
country: United Kingdom
facility: Panthera Biopartners
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
Study officials:
References: