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

Phase 3

Recruiting

• Conditions: Myasthenia Gravis, Ocular

• Registration Number: NCT06558279
• Lead Sponsor: argenx

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-08-14
• Study First Submitted QC: 2024-08-14
• Study First Posted: 2024-08-16
• Study Start: 2024-09-18
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-12
• Last Update Posted: 2025-06-15
• Primary Completion: 2025-12
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 124

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
MGII (PRO) ocular score change from baseline to day 29 in part A	Up to 29 days	The 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.

Secondary Outcome Measures

Name	Time	Method
Incidence of (serious) adverse events	Up to 111 weeks
MGII (PRO+PE) ocular score change from baseline to day 29 in part A	Up to 29 days	The 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 ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
MG-ADL ocular domain score change from baseline to day 29 in part A	Up to 29 days	The MG-ADL scale assesses MG symptoms and their effects on daily activities. The ocular score varies between 0 and 16 (with 16 the most severe).
MGII total score change from baseline to day 29 in part A	Up to 29 days	The MGII is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The score varies between 0 and 84. The higher the score, the more severe the disease.
MGII (PE) ocular score change from baseline to day 29 in part A	Up to 29 days	The MGII is a scoring tool measuring disease severity. It consists of 6 physical examinations (PE). The score varies between 0 and 5. The higher the score, the more severe the disease.
MG-ADL total score change from baseline to day 29 in part A	Up to 29 days	The MG-ADL scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
MGII ocular scores changes (PRO) from baseline in part A+B	Up to 111 weeks	The 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 ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
MG-QoL15r total score changes from baseline in part A+B	Up to 111 weeks	The MG-QoL15r questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life).
NEI VFQ-25 score changes from baseline in part A+B	Up to 111 weeks	The NEI VFQ-25 is a 25-item vision-targeted questionnaire. The score varies between 0 and 100 (with 0 being the worst score)
Percent change from baseline in total IgG levels over time in part A+B	Up to 111 weeks
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B	Up to 111 weeks
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B	Up to 111 weeks
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B	Up to 111 weeks

Trial Locations

Locations (59)

Northwestern Memorial Hospital; 🇺🇸 Chicago, Illinois, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

The Alfred Hospital; 🇦🇺 Melbourne, Australia

The First Affiliated Hospital of Chongqing Medical University; 🇨🇳 Chongqing, China

Fakultni nemocnice Brno; 🇨🇿 Brno, Czechia

Fondazione IRCCS Istituto Neurologico Carlo Besta; 🇮🇹 Milano, Italy

Saitama Medical Center; 🇯🇵 Kawagoe, Japan

Leids Universitair Medisch Centrum; 🇳🇱 Leiden, Netherlands

Hospital Universitario de Donostia; 🇪🇸 San Sebastián, Spain

Panthera Biopartners - Glasgow; 🇬🇧 Glasgow, United Kingdom

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