Myasthenia Gravis, Ocular
Conditions
Keywords
Myasthenia Gravis, Ocular myasthenia
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.
Interventions
COMBINATION_PRODUCTEfgartigimod PH20 SC
Subcutaneous efgartigimod PH20 SC given by prefilled syringe
OTHERPlacebo PH20 SC
Subcutaneous placebo PH20 SC given by prefilled syringe
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| MGII (PRO+PE) 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 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 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). |
| MGII total 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 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 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. |
| MG-ADL total score change from baseline to day 29 in part A | Up to 29 days | 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). |
| MGII ocular scores changes (PRO) from baseline in part A+B | Up to 111 weeks | 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. |
| Incidence of (serious) adverse events | Up to 111 weeks | — |
| MG-QoL15r total score changes from baseline in part A+B | Up to 111 weeks | 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). |
| NEI VFQ-25 score changes from baseline in part A+B | Up to 111 weeks | 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) |
| 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 | — |
Countries
Australia, Austria, Belgium, Canada, China, Cyprus, Czechia, Denmark, Finland, France, Georgia, Germany, Greece, Italy, Japan, Netherlands, Poland, Portugal, Serbia, South Korea, Spain, Sweden, United Arab Emirates, United Kingdom, United States
Outcome results
None listed