A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
- Conditions
- Myasthenia Gravis, Ocular
- Interventions
- Combination Product: Efgartigimod PH20 SCOther: Placebo PH20 SC
- 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 124
- 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
- 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
- Arm && Interventions
Group Intervention Description Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B Placebo PH20 SC Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B Efgartigimod PH20 SC in part A+B Efgartigimod PH20 SC Participants receiving efgartigimod PH20 SC during part A and part B Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B Efgartigimod PH20 SC Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B
- 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
Related Research Topics
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Trial Locations
- Locations (49)
Hôpital de la Citadelle
🇧🇪Liège, Belgium
Neurology Offices of South Florida
🇺🇸Boca Raton, Florida, United States
SFM Clinical Research, LLC
🇺🇸Boca Raton, Florida, United States
Neurology Associates PA
🇺🇸Maitland, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
National Neuromuscular Research Institute
🇺🇸Austin, Texas, United States
Box Hill Hospital
🇦🇺Box Hill, Australia
Gold Coast University Hospital
🇦🇺Southport, Australia
UZ Leuven
🇧🇪Leuven, Belgium
Maritime Neurology
🇨🇦Halifax, Canada
Ottawa Hospital Research Institute
🇨🇦Ottawa, Canada
Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital
🇨🇳Chengdu, China
Guangdong Province Traditional Chinese Medical Hospital
🇨🇳Guangzhou, China
Qilu Hospital of Shandong University
🇨🇳Jinan, China
Huashan Hospital Fudan University
🇨🇳Shanghai, China
Hospital Clinico San Carlos
🇪🇸Madrid, Spain
Cyprus Institute of Neurology and Genetics
🇨🇾Egkomi, Cyprus
Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice
🇨🇿Pardubice, Czech Republic
Aarhus Universitetshospital
🇩🇰Aarhus N, Denmark
Rigshospitalet
🇩🇰København, Denmark
Turun Yliopistollinen Keskussairaala
🇫🇮Turku, Finland
Petre Sarajishvili Institute of Neurology
🇬🇪Tbilisi, Georgia
New Hospitals
🇬🇪Tbilisi, Georgia
Pineo Medical Ecosystem
🇬🇪Tbilisi, Georgia
LLC Caucasus Medical Centre
🇬🇪Tbilisi, Georgia
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Chiba University Hospital
🇯🇵Chuo-Ku, Japan
Kyushu University Hospital
🇯🇵Fukuoka, Japan
General Hanamaki Hospital
🇯🇵Hanamaki-shi, Japan
Hiroshima University Hospital
🇯🇵Hiroshima, Japan
National Hospital Organization Utano National Hospital
🇯🇵Kyoto-Shi, Japan
IUHW Narita Hospital
🇯🇵Narita, Japan
Hospital Clinico Universitario de Valencia
🇪🇸Valencia, Spain
National Hospital Organization Hokkaido Medical Center
🇯🇵Sapporo-Shi, Japan
Tokyo Medical University Hospital
🇯🇵Shinjuku-Ku, Japan
Osaka University Hospital
🇯🇵Suita-Shi, Japan
Kyungpook National University Chilgok hospital
🇰🇷Daegu, Korea, Republic of
Korea University Anam Hospital
🇰🇷Seoul, Korea, Republic of
Severance Hospital Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
MICS Centrum Medyczne Bydgoszcz
🇵🇱Bydgoszcz, Poland
Centrum Medyczne Neurologia Slaska
🇵🇱Katowice, Poland
SP ZOZ Szpital Uniwersytecki
🇵🇱Kraków, Poland
Krakowska Akademia Neurologii
🇵🇱Kraków, Poland
CLINIREM Sp z o.o.
🇵🇱Lublin, Poland
University Clinical Center of Serbia
🇷🇸Belgrade, Serbia
University Clinical Center Nis
🇷🇸Nis, Serbia
Hospital Universitario Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital del Mar
🇪🇸Barcelona, Spain
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden