A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
NCT Number: NCT06558279
Acronym: ADAPT oculus
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE3
Conditions: Myasthenia Gravis, Ocular
Interventions: COMBINATION_PRODUCT: Efgartigimod PH20 SC|OTHER: Placebo PH20 SC
Sponsor: argenx
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 124
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-09-15
Completion Date: 2027-12

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.

Primary Outcome Measures: MGII (PRO) ocular score change from baseline to day 29 in part A, 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., Up to 29 days
Secondary Outcome Measures: MGII (PRO+PE) ocular score change from baseline to day 29 in part A, 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., Up to 29 days|MG-ADL ocular domain score change from baseline to day 29 in part A, 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)., Up to 29 days|MGII total score change from baseline to day 29 in part A, 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., Up to 29 days|MGII (PE) ocular score change from baseline to day 29 in part A, 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., Up to 29 days|MG-ADL total score change from baseline to day 29 in part A, 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)., Up to 29 days|MGII ocular scores changes (PRO) from baseline in part A+B, 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., Up to 111 weeks|Incidence of (serious) adverse events, Up to 111 weeks|MG-QoL15r total score changes from baseline in part A+B, 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)., Up to 111 weeks|NEI VFQ-25 score changes from baseline in part A+B, The NEI VFQ-25 is a 25-item vision-targeted questionnaire. The score varies between 0 and 100 (with 0 being the worst score), Up to 111 weeks|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