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

NCT06558279 | Active Not Recruiting Phase 3 FDA Drug

• 2 other identifiers: ARGX-113-23152024-514133-38-00
• Study Type: interventional
• Target: 141
• Locations: 23 countries
• Sites: 100

Brief 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.

Conditions

Myasthenia Gravis, Ocular

Keywords

Myasthenia Gravis; Ocular myasthenia

Outcome Measures

Primary Outcomes (1)

• MGII (PRO) ocular score change from baseline to day 29 in part A

  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.

  Up to 29 days

Secondary Outcomes (13)

• MGII (PRO+PE) ocular score change from baseline to day 29 in part A

  Up to 29 days

• MG-ADL ocular domain score change from baseline to day 29 in part A

  Up to 29 days

• MGII total score change from baseline to day 29 in part A

  Up to 29 days

• MGII (PE) ocular score change from baseline to day 29 in part A

  Up to 29 days

• MG-ADL total score change from baseline to day 29 in part A

  Up to 29 days

Study Arms (2)

Efgartigimod PH20 SC in part A+B

EXPERIMENTAL

Participants receiving efgartigimod PH20 SC during part A and part B

Combination Product: Efgartigimod PH20 SC

Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B

EXPERIMENTAL

Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B

Combination Product: Efgartigimod PH20 SC; Other: Placebo PH20 SC

Interventions

Placebo PH20 SC OTHER

Subcutaneous placebo PH20 SC given by prefilled syringe

Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B

Efgartigimod PH20 SC COMBINATION_PRODUCT

Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Efgartigimod PH20 SC in part A+B; Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• argenx: lead

Study Sites (102)

HonorHealth Neurology - Bob Bové Neuroscience Institute

Scottsdale, Arizona, 85251, United States

Location

Loma Linda University Health

Loma Linda, California, 92354, United States

Location

USC Roski Eye Institute - Los Angeles

Los Angeles, California, 90033, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Neurology Offices of South Florida

Boca Raton, Florida, 33428-4221, United States

Location

SFM Clinical Research, LLC

Boca Raton, Florida, 33487-2768, United States

Location

Neurology Associates PA

Maitland, Florida, 32751, United States

Location

University of South Florida

Tampa, Florida, 33620, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

University of California Irvine

Columbia, Maryland, 21044, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Duke Neurological Disorders Clinic

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Martha Morehouse Medical plaza

Columbus, Ohio, 43221, United States

Location

Penn Medicine University City

Philadelphia, Pennsylvania, 19104, United States

Location

National Neuromuscular Research Institute

Austin, Texas, 78759-8402, United States

Location

Houston Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

Box Hill Hospital

Box Hill, 3128, Australia

Location

The Alfred Hospital

Melbourne, 3004, Australia

Location

Gold Coast University Hospital

Southport, 4215, Australia

Location

Universitätsklinikum AKH Wien

Vienna, 1090, Austria

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Hôpital de la Citadelle

Liège, 4000, Belgium

Location

Maritime Neurology

Halifax, B3R 1V9, Canada

Location

London Health Sciences Centre

London, N6A 5A5, Canada

Location

Centre Hospitalier de l'Universite de Montreal

Montreal, H2X 0A9, Canada

Location

Ottawa Hospital Research Institute

Ottawa, K1Y 4E9, Canada

Location

Hopital de L'enfant Jesus

Québec, G1J 1Z4, Canada

Location

Toronto General Hospital

Toronto, M5G 2C4, Canada

Location

Xiangya Hospital Central South University

Changsha, 410008, China

Location

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

Chengdu, 610072, China

Location

The First Affiliated Hospital of Chongqing Medical University

Chongqing, 400016, China

Location

Fujian Medical University Union Hospital

Fuzhou, 350001, China

Location

Guangdong Province Traditional Chinese Medical Hospital

Guangzhou, 510120, China

Location

The First Affiliated Hospital of Guangzhou University Chinese Medicine

Guangzhou, 510405, China

Location

Qilu Hospital of Shandong University

Jinan, 250012, China

Location

The First Affiliated Hospital of Nanchang University - Xianghu Campus

Nanchang, 330000, China

Location

Huashan Hospital Fudan University

Shanghai, 200040, China

Location

Shijiazhuang People's Hospital - Fangbeilu Campus

Shijiazhuang, 050011, China

Location

Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, 430030, China

Location

Renmin Hospital of Wuhan University - Main Campus

Wuhan, 430060, China

Location

Cyprus Institute of Neurology and Genetics

Égkomi, 2371, Cyprus

Location

Fakultni nemocnice Brno

Brno, 625 00, Czechia

Location

Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice

Pardubice, 532 03, Czechia

Location

Aarhus Universitetshospital

Aarhus N, 8200, Denmark

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Turun Yliopistollinen Keskussairaala

Turku, 20521, Finland

Location

Hôpital Fondation Adolphe de Rothschild

Paris, 75019, France

Location

Petre Sarajishvili Institute of Neurology

Tbilisi, 112, Georgia

Location

New Hospitals

Tbilisi, 114, Georgia

Location

Pineo Medical Ecosystem

Tbilisi, 114, Georgia

Location

LLC Caucasus Medical Centre

Tbilisi, 186, Georgia

Location

Charité - Universitätsmedizin Berlin

Berlin, 13353, Germany

Location

Universität Georg August

Göttingen, 37075, Germany

Location

Eginitio Hospital

Athens, 115 28, Greece

Location

University General Hospital of Patras

Pátrai, 265 04, Greece

Location

ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII

Bergamo, 24127, Italy

Location

IRCCS Istituto dell Scienze Neurologiche di Bologna

Bologna, 40139, Italy

Location

Ospedale San Raffaele S.r.l.

Milan, 20132, Italy

Location

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

Location

AORN Antonio Cardarelli

Naples, 80131, Italy

Location

Azienda Ospedaliero-Universitaria Sant'Andrea

Roma, 00189, Italy

Location

Chiba University Hospital

Chūōku, 260-8677, Japan

Location

Kyushu University Hospital

Fukuoka, 812-8582, Japan

Location

General Hanamaki Hospital

Hanamaki-shi, 025-0075, Japan

Location

Hiroshima University Hospital

Hiroshima, 734-8551, Japan

Location

Saitama Medical Center

Kawagoe, 350-0844, Japan

Location

National Hospital Organization Utano National Hospital

Kyoto, 616-8255, Japan

Location

IUHW Narita Hospital

Narita, 286-8520, Japan

Location

National Hospital Organization Hokkaido Medical Center

Sapporo, 063-0005, Japan

Location

Kindai University Hospital

Sayama, 589-8511, Japan

Location

Tokyo Medical University Hospital

Shinjuku-Ku, 160-0023, Japan

Location

Osaka University Hospital

Suita-Shi, 565-0871, Japan

Location

Leids Universitair Medisch Centrum

Leiden, 2333 ZA, Netherlands

Location

MICS Centrum Medyczne Bydgoszcz

Bydgoszcz, 85-065, Poland

Location

Centrum Medyczne Neurologia Slaska

Katowice, 40-689, Poland

Location

SP ZOZ Szpital Uniwersytecki

Krakow, 31-503, Poland

Location

Krakowska Akademia Neurologii

Krakow, 31-505, Poland

Location

CLINIREM Sp z o.o.

Lublin, 20-064, Poland

Location

ULS de Santa Maria, EPE - Hospital de Santa Maria

Lisbon, 1649-035, Portugal

Location

ULS de Santo António, EPE - Hospital de Santo António

Porto, 4099-001, Portugal

Location

University Clinical Center of Serbia

Belgrade, 11000, Serbia

Location

University Clinical Center Nis

Niš, 18 000, Serbia

Location

Kyungpook National University Chilgok hospital

Daegu, 41404, South Korea

Location

Korea University Anam Hospital

Seoul, 2841, South Korea

Location

Severance Hospital Yonsei University Health System

Seoul, 3722, South Korea

Location

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital del Mar

Barcelona, 8003, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28304, Spain

Location

Hospital Universitario de Donostia

San Sebastián, 20014, Spain

Location

Hospital Clinico Universitario de Valencia

Valencia, 46010, Spain

Location

Sahlgrenska Universitetssjukhuset

Gothenburg, 413 45, Sweden

Location

Mediclinic Parkview

Dubai, 51122, United Arab Emirates

Location

Royal Sussex County Hospital

Brighton, BN2 5BE, United Kingdom

Location

Panthera Biopartners - Glasgow

Glasgow, G51 4TY, United Kingdom

Location

Leeds General Infirmary

Leeds, LS1 3EX, United Kingdom

Location

Dementech Neurosciences Clinical Academic Centre

London, W1G 9ST, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2024
• First Posted: August 16, 2024
• Study Start: September 18, 2024
• Primary Completion: January 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: December 15, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

DK (2); GE (4); PT (2); PL (5); CZ (2); FR (1); US (20); ES (6); AE (1); IT (6); SE (2); GB (5); BE (2); DE (2); CN (12); AU (3); CY (1); NL (1); GR (2); KR (3); JP (11); CA (6); FI (1)