A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis

NCT06558279 · tracked via the Priya Life Science UK tracker

Sponsor

argenx

Phase

Phase 3

Started

2024-09-18

Last updated

2026-07-02

Condition(s) studied

Myasthenia Gravis, Ocular

Investigational drug(s) / intervention(s)

Efgartigimod PH20 SCPlacebo PH20 SC

Efgartigimod PH20 SC: Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Placebo PH20 SC: Subcutaneous placebo PH20 SC given by prefilled syringe

Study summary

The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Is at least 18 years of age and the local legal age of consent for clinical studies * Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy" * Is MGFA Class I (any ocular muscle weakness) * Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2 Exclusion Criteria: * Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia * Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk

Primary outcome measure(s)

MGII (PRO) ocular score change from baseline to day 29 in part A — Up to 29 days
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.

Trial sites (102)

Facility	City	Region
HonorHealth Neurology - Bob Bové Neuroscience Institute	Scottsdale	Arizona
Loma Linda University Health	Loma Linda	California
USC Roski Eye Institute - Los Angeles	Los Angeles	California
University of California San Francisco	San Francisco	California
Neurology Offices of South Florida	Boca Raton	Florida
SFM Clinical Research, LLC	Boca Raton	Florida
Neurology Associates PA	Maitland	Florida
University of South Florida	Tampa	Florida
Northwestern Memorial Hospital	Chicago	Illinois
University of California Irvine	Columbia	Maryland
Massachusetts General Hospital	Boston	Massachusetts
Henry Ford Health System	Detroit	Michigan
Robert Wood Johnson Medical School	New Brunswick	New Jersey
University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
Duke Neurological Disorders Clinic	Durham	North Carolina
Cleveland Clinic	Cleveland	Ohio
Martha Morehouse Medical plaza	Columbus	Ohio
Penn Medicine University City	Philadelphia	Pennsylvania
National Neuromuscular Research Institute	Austin	Texas
Houston Methodist Neurological Institute	Houston	Texas
Box Hill Hospital	Box Hill	Australia
The Alfred Hospital	Melbourne	Australia
Gold Coast University Hospital	Southport	Australia
Universitätsklinikum AKH Wien	Vienna	Austria
UZ Leuven	Leuven	Belgium
Hôpital de la Citadelle	Liège	Belgium
Maritime Neurology	Halifax	Canada
London Health Sciences Centre	London	Canada
Centre Hospitalier de l'Universite de Montreal	Montreal	Canada
Ottawa Hospital Research Institute	Ottawa	Canada
Hopital de L'enfant Jesus	Québec	Canada
Toronto General Hospital	Toronto	Canada
Xiangya Hospital Central South University	Changsha	China
Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital	Chengdu	China
The First Affiliated Hospital of Chongqing Medical University	Chongqing	China
Fujian Medical University Union Hospital	Fuzhou	China
Guangdong Province Traditional Chinese Medical Hospital	Guangzhou	China
The First Affiliated Hospital of Guangzhou University Chinese Medicine	Guangzhou	China
Qilu Hospital of Shandong University	Jinan	China
The First Affiliated Hospital of Nanchang University - Xianghu Campus	Nanchang	China

+ 62 more sites — see the full list on the official registry below.

More argenx trials in the UK

A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis Recruiting

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis Active, not recruiting

A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren's Syndrome Active, not recruiting

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 Recruiting

A Clinical Trial to Investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy Active, not recruiting

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 NCT06558279 on ClinicalTrials.gov ↗ ← All trials in the UK