A Phase 2a Randomized, Double-blind, Placebo-controlled, Parallel Groups, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALXN1830 Administered Subcutaneously in Adult Patients With Generalized Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- AEs And SAEs Up To Week 82
研究概览
简要总结
This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).
详细描述
Participants have the option to enroll in an Open-label Extension (OLE) Period to receive ALXN1830 up to 58 weeks (including follow-up period).
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
- Double (Participant, Investigator)
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
- •Diagnosis of myasthenia gravis.
- •Positive serologic test for anti-acetylcholine receptor antibodies.
- •Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening.
- •MG-ADL profile must be ≥
- •Participants receiving stable treatment with azathioprine; other immunosuppressive therapies.
- •Total IgG level at Screening ≥ 600 milligrams/deciliter.
排除标准
- •History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening.
- •Any untreated thymic malignancy, carcinoma, or thymoma.
- •Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1).
- •Use of rituximab within the 3 months (90 days) prior to Screening.
- •Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer).
- •Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
研究组 & 干预措施
ALXN1830 Dosing Arm 1
Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.
干预措施: ALXN1830 (Drug)
ALXN1830 Dosing Arm 2
Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.
干预措施: ALXN1830 (Drug)
ALXN1830 Dosing Arm 3
Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.
干预措施: ALXN1830 (Drug)
ALXN1830 Dosing Arm 3
Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.
干预措施: Placebo (Other)
结局指标
主要结局
AEs And SAEs Up To Week 82
时间窗: Up to Week 82 (OLE)
Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24
时间窗: Up to Week 24
Change From Baseline In Serum Total Immunoglobulin G (IgG)
时间窗: Up to Week 24
次要结局
- Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks(Up to Week 24)
- Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks(Up to Week 8)
- Titers Of ADA And Nab Against ALXN1830(Up to Week 24)
- Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score(Up to Week 24)
- Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830(Up to Week 24)
- Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score(Up to Week 24)
- Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score(Up to Week 24)
- Serum Trough Concentrations Of ALXN1830(Up to Week 24)
- Change From Baseline In IgG Subtypes(Up to Week 24)