A Phase 2, Randomized, Double-Blind, Placebo-Controlled Multicenter Study to Evaluate the Efficacy and Safety of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- Percentage Of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction Of ≥ 2 Points In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy
研究概览
简要总结
This study will evaluate the efficacy and safety of ALXN2050 (120 milligrams [mg], 180 mg) in participants with generalized myasthenia gravis (gMG).
Safety will be monitored throughout the study.
详细描述
The study consists of a blinded 8-week Primary Evaluation Period (PEP) and a blinded 26-week Extended Treatment Period (ETP). After completion of 34 weeks of treatment, participants will enter an Open-label Extension (OLE) Period for up to 1.5 years.
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
Masking of treatment allocation will be observed until at least Week 34.
入排标准
- 年龄范围
- 18 Years 至 130 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit. Confirmation of MG must be made via the following:
- •Positive serologic test for anti AChR antibodies at the Screening Visit, and
- •Abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, or
- •Positive response to an AChEI test (eg, edrophonium chloride test), or
- •Improvement of signs or symptoms related to MG during treatment with an oral AChEI, as determined by the treating physician
- •Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit.
- •MG-ADL total score must be ≥ 5 (with at least 50% of the score attributed to non-ocular elements) at the Screening Visit and at randomization (Day 1).
- •Participants receiving treatment with protocol-specified immunosuppressive therapies, corticosteroids, or acetylcholinesterase inhibitors must have been receiving treatment and on a stable dose prior to the date of the Screening Visit, with no changes to the regimen expected during screening, the PEP, and/or the ETP.
排除标准
- •Estimated glomerular filtration rate ≤ 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration.
- •History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to the Screening Visit.
- •Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet pre-specified conditions outlined in the protocol.
- •Clinical features consistent with Clinical Deterioration at the time of the Screening Visit or at any time during the Screening Period prior to randomization (Day 1).
- •Use of the following within the time periods specified below:
- •Intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit.
- •Use of tacrolimus or cyclosporine within the 4 weeks (28 days) prior to the Screening Visit.
研究组 & 干预措施
ALXN2050: 180 mg
Participants will receive ALXN2050.
干预措施: ALXN2050 (Drug)
ALXN2050: 120 mg
Participants will receive ALXN2050.
干预措施: ALXN2050 (Drug)
Placebo
Participants will receive placebo followed by ALXN2050.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage Of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction Of ≥ 2 Points In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy
时间窗: Baseline through Week 8
The MG-ADL profile is an 8-item participant-reported scale that focuses on relevant symptoms and functional performance of ADL in participants with MG. The 8 items of the MG-ADL 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 of MG. Each response is graded 0 (normal) to 3 (most severe). The MG-ADL total score was calculated as the sum of the scores of the 8 items and ranges from 0 to 24, with higher scores indicating worse function.
次要结局
- Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8(Baseline, Week 8)
- Change From Baseline In MG-ADL Total Score At Week 8(Baseline, Week 8)
- Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8(Baseline, Week 8)