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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
141
Subcutaneous efgartigimod PH20 SC given by prefilled syringe
Subcutaneous placebo PH20 SC given by prefilled syringe
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.
Time frame: Up to 29 days
MGII (PRO+PE) 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 ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
Time frame: Up to 29 days
MG-ADL ocular domain score change from baseline to day 29 in part A
The Myasthenia Gravis Activities of Daily Living (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).
Time frame: Up to 29 days
MGII total 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 score varies between 0 and 84. The higher the score, the more severe the disease.
Time frame: Up to 29 days
MGII (PE) 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 6 physical examinations (PE). The score varies between 0 and 5. The higher the score, the more severe the disease.
Time frame: Up to 29 days
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HonorHealth Neurology - Bob Bové Neuroscience Institute
Scottsdale, Arizona, United States
Loma Linda University Health
Loma Linda, California, United States
USC Roski Eye Institute - Los Angeles
Los Angeles, California, United States
University of California San Francisco
San Francisco, California, United States
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
Northwestern Memorial Hospital
Chicago, Illinois, United States
University of California Irvine
Columbia, Maryland, United States
...and 92 more locations
MG-ADL total score change from baseline to day 29 in part A
The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
Time frame: Up to 29 days
MGII ocular scores changes (PRO) from baseline in part A+B
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 ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
Time frame: Up to 111 weeks
Incidence of (serious) adverse events
Time frame: Up to 111 weeks
MG-QoL15r total score changes from baseline in part A+B
The Myasthenia Gravis Quality of Life 15-item scale revised (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).
Time frame: Up to 111 weeks
NEI VFQ-25 score changes from baseline in part A+B
The 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) is a 25-item vision-targeted questionnaire. The score varies between 0 and 100 (with 0 being the worst score)
Time frame: Up to 111 weeks
Percent change from baseline in total IgG levels over time in part A+B
Time frame: Up to 111 weeks
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B
Time frame: Up to 111 weeks
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B
Time frame: Up to 111 weeks
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B
Time frame: Up to 111 weeks