A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 3, Pivotal Study With an Open-Label Extension Period to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-Associated Disease (MOG-AD)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 113
- 试验地点
- 138
- 主要终点
- For Part A: Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period
研究概览
简要总结
The purpose of the study is to evalute the efficacy, safety and tolerability of rozanolixizumab for treatment of adult participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOGAD).
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
MOG001 consists of a Double-Blind (Part A) and an Open-Label Extension Study Period (Part B).
入排标准
- 年龄范围
- 18 Years 至 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be ≥18 to ≤89 years of age, at the time of signing the informed consent
- •Confirmed diagnosis of MOGAD consistent with published diagnostic criteria for MOGAD
- •Participant has history of relapsing MOGAD with at least 1 documented relapse over the last 12 months and a documented positive serum MOG Ab test using a cell-based assay (CBA) within 6 months prior to randomization
- •Participant must be clinically stable at the time of the Screening Visit and during the Screening Period
排除标准
- •Participant has been diagnosed with a neurological autoimmune disease (including multiple sclerosis (MS) and aquaporin-4 positive neuromyelitis optica spectrum disorder (NMOSD)), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant
- •Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator, including participants with a serious infection within 6 weeks prior to the first dose of the investigational medicinal product (IMP)
- •Participant has a current or medical history of primary immunodeficiency
- •Participant tests positive for aquaporin-4 antibodies at Screening
- •Participant has a serum total IgG level ≤ 5.5g/L
研究组 & 干预措施
Rozanolixizumab Arm
Participants randomized into this arm will receive rozanolixizumab at pre-specified timepoints.
干预措施: Rozanolixizumab (Drug)
Placebo Arm
Participants randomized into this arm will receive placebo at pre-specified timepoints to maintain the blinding.
干预措施: Placebo (Other)
结局指标
主要结局
For Part A: Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period
时间窗: Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks)
The TTFR (days) will be defined as the interval between the date of randomization and the first date of the objective relapse. During the Double Blind (DB) Treatment Period (Part A); EDB/EWD = End of Double-Blind/Early Withdrawal Visit
For Part B: Incidence of treatment-emergent adverse events (TEAEs) during OLE Treatment Period
时间窗: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Open-Label Extension (OLE) Treatment Period (Part B); EOS/EWD = End of Study/Early Withdrawal Visit.
For Part B: Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period
时间窗: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs leading to discontinuation of the study are reported.
次要结局
- For Part A: Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period(Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks))
- For Part B: Independently centrally adjudicated annualized relapse rate (ARR) during the DB and OLE Treatment Period(Baseline (Week 1) to EOS/EWD Visit (up to OLE Week 52))
- For Part A: Incidence of treatment-emergent adverse events (TEAEs) during the DB Treatment Period(Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks))
- For Part A: Change from Baseline in Low-Contrast Monocular Visual Acuity (Worst Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the EDB/EWD Visit(From Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks))
- For Part A: Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the EDB/EWD Visit (with confirmation at 3 months)(Baseline (Week 1), EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks))