A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- argenx
- 入组人数
- 141
- 试验地点
- 197
- 主要终点
- MGII (PRO) ocular score change from baseline to day 29 in part A
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Efgartigimod PH20 SC in part A+B
Participants receiving efgartigimod PH20 SC during part A and part B
干预措施: Efgartigimod PH20 SC (Combination Product)
Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B
Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B
干预措施: Placebo PH20 SC (Other)
Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B
Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B
干预措施: Efgartigimod PH20 SC (Combination Product)
结局指标
主要结局
MGII (PRO) ocular score change from baseline to day 29 in part A
时间窗: 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.
MGII (PRO) ocular score change from baseline to day 29 in part A
时间窗: 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.
次要结局
- MG-ADL total score change from baseline to day 29 in part A(Up to 29 days)
- MGII (PRO+PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
- MG-ADL ocular domain score change from baseline to day 29 in part A(Up to 29 days)
- MGII total score change from baseline to day 29 in part A(Up to 29 days)
- MGII (PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
- MGII ocular scores changes (PRO) from baseline in part A+B(Up to 111 weeks)
- Incidence of (serious) adverse events(Up to 111 weeks)
- MG-QoL15r total score changes from baseline in part A+B(Up to 111 weeks)
- NEI VFQ-25 score changes from baseline in part A+B(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)
- MGII (PRO+PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
- MG-ADL ocular domain score change from baseline to day 29 in part A(Up to 29 days)
- MGII total score change from baseline to day 29 in part A(Up to 29 days)
- MGII (PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
- MG-ADL total score change from baseline to day 29 in part A(Up to 29 days)
- MGII ocular scores changes (PRO) from baseline in part A+B(Up to 111 weeks)
- Incidence of (serious) adverse events(Up to 111 weeks)
- MG-QoL15r total score changes from baseline in part A+B(Up to 111 weeks)
- NEI VFQ-25 score changes from baseline in part A+B(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)