A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 175
- 试验地点
- 1
- 主要终点
- Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26
研究概览
简要总结
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
All investigative site personnel, sponsor staff, sponsor designees, staff directly associated with the conduct of the study, and all participants will be blinded to treatment assignments.
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
- •Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
- •MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
- •Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).
排除标准
- •Medical Conditions
- •Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening.
- •History of thymectomy within the 12 months prior to screening.
- •History of N meningitidis infection.
- •Use of the following within the time period specified below:
- •IV immunoglobulin within 4 weeks of randomization
- •Use of plasma exchange within 4 weeks of randomization
- •Use of rituximab within 6 months of screening
- •Participants who have received previous treatment with complement inhibitors (for example, eculizumab).
研究组 & 干预措施
Ravulizumab
Participants will receive ravulizumab for the duration of the study.
干预措施: Ravulizumab (Biological)
Placebo
Participants will receive placebo during the 26-week randomized-controlled period of the study, after which they will enter the open-label extension period of the study and receive ravulizumab.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26
时间窗: Baseline, Week 26
The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Estimates were based on Mixed Effect Repeated Measures (MMRM) that included treatment group, stratification factor region, and MG-ADL total score at baseline, study visit, and study visit by treatment group interaction.
次要结局
- Percentage of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26(Week 26)
- Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26(Week 26)
- Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26(Baseline, Week 26)
- Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26(Baseline, Week 26)
- Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26(Baseline, Week 26)