A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 43
- 试验地点
- 20
- 主要终点
- Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9
研究概览
简要总结
The purpose of the study is to evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe.
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)
盲法说明
This is an Investigator- and Subject-Blind study.
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
- •Subject has a well-documented diagnosis of myasthenia gravis (MG) at Visit 1 (Screening), based on subject history and supported by previous evaluations
- •Subject would currently be considered for treatment with immunological therapy (immunoglobulin/plasma exchange (IVIG/PLEX)) by the investigator
- •Subject has a well-documented record of autoantibodies against anti-acetylcholine receptor (Anti-AChR) or anti-muscle specific kinase (Anti-MuSK) prior to Screening
- •Female subjects must either be: postmenopausal, permanently sterilized or if childbearing potential applicable will use a highly effective method of birth control
- •Male subjects must be willing to use a method of contraception
排除标准
- •Subject has previously received treatment in this study or subject has previously been exposed to UCB7665
- •Subject has participated in another study of an investigational medicinal product (IMP; or a medical device) within the previous 30 days of Screening or is currently participating in another study of an investigational medicinal product (IMP; or a medical device)
- •Subject has a known hypersensitivity to any components of the IMP
- •Subject has a history of hyperprolinemia, since L-proline is a constituent of the UCB7665 IMP
- •Subjects with Myasthenia Gravis (MG) only affecting the ocular muscles
- •Subjects with severe weakness affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening or impending crisis
- •Subject has quantitative myasthenia gravis (QMG) score of <11 at Baseline
- •Subject has a serum total immunoglobulin G (IgG) level <= 6g/L at Screening
- •Absolute neutrophil count <1500 cells/mm^3
- •Subject has any medical condition (acute or chronic illness) or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject's ability to participate in this study
- •Subject has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or would compromise the subject's ability to participate in this study
- •Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP
- •Subject has received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline (whichever is longer)
研究组 & 干预措施
Dosage Regimen 2
Subjects randomized in dosage regimen 2 will receive 3 doses of placebo in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
干预措施: Placebo (Other)
Dosage Regimen 2
Subjects randomized in dosage regimen 2 will receive 3 doses of placebo in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
干预措施: UCB7665 (Drug)
Dosage Regimen 1
Subjects randomized in dosage regimen 1 will receive 3 doses of UCB7655 (dose 1) in dosing period 1 and will then be re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
干预措施: UCB7665 (Drug)
结局指标
主要结局
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9
时间窗: From Baseline to Visit 9 (up to Day 29)
The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.
次要结局
- Change From Baseline in Myasthenia Gravis-Composite Score to Visit 9(From Baseline to Visit 9 (up to Day 29))
- Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) Score to Visit 9(From Baseline to Visit 9 (up to Day 29))