NCT06706622进行中（未招募）3 期
Interventional, Randomized, Double-blind, Placebo-controlled, Optional Open-label Extension Trial of Lu AF82422 in Participants With Multiple System Atrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 401
- 试验地点
- 138
- 主要终点
- Rest of the World (RoW; All Countries Except European Union [EU] and Japan [JP]) Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
研究概览
简要总结
The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA).
详细描述
This study will consist of a screening period of 10 days up to 6 weeks, a 72-week placebo-controlled period (PCP), and will include a 72-week optional open-label treatment extension (OLE) period. Participants in the PCP will be randomized to amlenetug or placebo. All participants entering the OLE will receive amlenetug during the OLE. Participants will receive intravenous infusions approximately every 4 weeks during both the PCP and OLE.
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)
入排标准
- 年龄范围
- 40 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant has a diagnosis of clinically established multiple system atrophy parkinsonian type (MSA-P) or multiple system atrophy cerebellar type (MSA-C), or clinically probable MSA-P or MSA-C, according to the 2022 Movement Disorders Society (MDS) criteria for the diagnosis of MSA at the Screening Visit.
- •The participant had onset of motor MSA symptoms (that is, parkinsonian and/or cerebellar) within 5 years prior to the Screening Visit in the judgement of the investigator.
- •The participant has an anticipated survival of >3 years, in the opinion of the investigator, at the Screening Visit.
- •The participant has suitable peripheral venous access for investigational medicinal product (IMP) administration and blood sampling.
- •The participant has an UMSARS Part I score ≤16 (omitting item 11 on sexual function) at the Screening Visit.
排除标准
- •The participant has previously been dosed with amlenetug.
- •The participant has taken any active IMP within 3 months or 5 half lives of that product, whichever is longer, prior to the first dose of IMP.
- •The participant has 2 or more first degree relatives with a history of MSA.
- •The participant, if of MSA-P subtype, has unexplained anosmia (not explained by other common causes such as allergic rhinitis or smoking, nasal structural lesions, or nasal surgery) on olfactory testing at the Screening Visit.
- •The participant has evidence (clinically or on magnetic resonance imaging (MRI)) and/or history of any clinically significant disease or condition other than MSA, that is, in the investigator's opinion, likely to affect CNS functioning, e.g., serious neurological disorder, other intracranial or systemic disease.
- •The participant has a current diagnosis of movement disorders that could mimic MSA, e.g., Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, pharmacological, or post-encephalitic parkinsonism, per investigator discretion. Participants who have previously been incorrectly diagnosed with Parkinson's disease will not be excluded.
- •Other protocol-defined inclusion and exclusion criteria apply.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive commercially available saline solution for infusion
干预措施: Placebo (Drug)
Amlenetug
Experimental
Participants will receive amlenetug by intravenous infusion
干预措施: Amlenetug (Drug)
结局指标
主要结局
Rest of the World (RoW; All Countries Except European Union [EU] and Japan [JP]) Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
时间窗: Baseline up to Week 72
Mortality-adjusted clinical progression will be assessed by the composite endpoint modified Unified Multiple System Atrophy Rating Scale (mUMSARS) score and time-to-death (any cause).
EU and JP Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
时间窗: Baseline up to Week 72
Mortality-adjusted clinical progression will be assessed by the composite endpoint Unified Multiple System Atrophy Rating Scale (UMSARS) Total Score (TS) and time-to-death (any cause).
次要结局
- Change from Baseline in Clinical Global Impression - Severity of Illness (CGI-S) Score(Baseline, Week 72)
- Change from Baseline in Patient Global Impression - Severity of Illness (PGI-S) Score(Baseline, Week 72)
- Change from Baseline in Observer-reported Global Impression-Severity of Illness (OGI-S) Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part IV Score(Baseline, Week 72)
- Change from Baseline in Schwab and England Activities of Daily Living (SE-ADL) Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part I Item 1: Speech Score(Baseline, Week 72)
- Change from Baseline in EuroQol 5-dimensions, 5-levels (EQ-5D-5L) Domain Score(Baseline, Week 72)
- Change from Baseline in EQ-5D-5L Visual Analog Scale (VAS) Score(Baseline, Week 72)
- Change from Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Questionnaire Domain Scores(Baseline, Week 72)
- Combined Clinical Progression and Survival Score(Baseline, Week 72)
- Time from Baseline to Death (Any Cause)(Baseline up to Week 72)
- Percentage Change from Baseline in Brain Volume in Brain Regions of Interest (ROIs)(Baseline, Week 72)
- Number of Participants with Treatment-emergent Adverse Events (TEAEs)(Day 1 up to Week 144)
- EU and JP Regional-specific Outcome Measure: Change from Baseline in UMSARS TS(Baseline, Week 72)
- Change from Baseline in Observer-reported Global Impression-Severity of Illness (OGI-S) Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part IV Score(Baseline, Week 72)
- Change from Baseline in Schwab and England Activities of Daily Living (SE-ADL) Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part I Item 1: Speech Score(Baseline, Week 72)
- Time to Disability(Baseline up to Week 72)
- RoW Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression(Baseline up to Week 72)
- RoW Regional-specific Outcome Measure: Change from Baseline in UMSARS TS(Baseline, Week 72)
- Change from Baseline in EQ-5D-5L Visual Analog Scale (VAS) Score(Baseline, Week 72)
- Change from Baseline in mUMSARS Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part I Score(Baseline, Week 72)
- Change from Baseline in UMSARS Part II Score(Baseline, Week 72)
- Change from Baseline in Clinical Global Impression - Severity of Illness (CGI-S) Score(Baseline, Week 72)
- Change from Baseline in Patient Global Impression - Severity of Illness (PGI-S) Score(Baseline, Week 72)
- Change from Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Questionnaire Domain Scores(Baseline, Week 72)
- Time from Baseline to Death (Any Cause)(Baseline up to Week 72)
- Number of Participants with an Absolute Increase in UMSARS TS of <16, <21, and <26 Points(Baseline to Week 72)
- Change from Baseline in EuroQol 5-dimensions, 5-levels (EQ-5D-5L) Domain Score(Baseline, Week 72)
- Percentage Change from Baseline in Brain Volume in Brain Regions of Interest (ROIs)(Baseline, Week 72)
- Combined Clinical Progression and Survival Score(Baseline, Week 72)
- Number of Participants with an Absolute Increase in mUMSARS Score of <5, <7, and <9 points(Baseline to Week 72)
- Number of Participants with Anti-amlenetug Antibodies (ADAs)(Baseline up to Week 144)
- Area Under the Curve (AUC) of amlenetug(Baseline up to Week 72)
- Maximum Observed Concentration (Cmax) of Amlenetug(Baseline up to Week 72)
- Number of Participants with Treatment-emergent Adverse Events (TEAEs)(Day 1 up to Week 144)
- Mortality-adjusted Clinical Progression(Baseline up to Week 72)
研究者
研究点 (138)
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相关资讯
Lundbeck Completes Early Patient Enrollment in Phase 3 Trial for Rare Neurodegenerative Disease MSA- Lundbeck announced ahead-of-schedule completion of patient randomization in the global Phase 3 MASCOT trial evaluating amlenetug for multiple system atrophy (MSA), a rare and fatal neurodegenerative disorder with no approved treatments.
- Amlenetug is a novel monoclonal antibody targeting α-synuclein protein that showed promising results in Phase 2 trials and could become the first-in-class therapy for MSA.
- The early enrollment completion reflects strong engagement from the global MSA community and underscores the urgent unmet medical need for this devastating disease.
- Headline results are anticipated in Q3 2027 with expected market launch in Q1 2029, offering hope for patients with this rapidly progressing condition.6 months agoLundbeck Advances Innovative Phase 3 Trial Design for Amlenetug in Multiple System Atrophy- Lundbeck will present details of its Phase 3 MASCOT trial for amlenetug, a first-in-class monoclonal antibody targeting α-synuclein aggregation in Multiple System Atrophy patients.
- The innovative trial design employs Bayesian progression modeling methods to address unique challenges in rare disease drug development with limited patient populations.
- Multiple System Atrophy is a rapidly progressing neurodegenerative disease with no approved treatments, affecting patients who typically live 6-9 years after symptom onset.
- Amlenetug has received Orphan Drug Designation from FDA and EMA, plus SAKIGAKE designation in Japan, highlighting its potential therapeutic significance.11 months agoLundbeck Presents Phase II Data for Amlenetug in Multiple System Atrophy at International Congress- Lundbeck presented results from the AMULET phase II trial investigating amlenetug, a monoclonal antibody targeting α-synuclein, as a potential treatment for multiple system atrophy (MSA).
- The company shared new insights from the TALISMAN natural history study, providing critical data on early disease progression in MSA patients to support phase III development.
- Patient perspectives from the AMULET trial informed the design of the upcoming phase III MASCOT trial, demonstrating Lundbeck's commitment to patient-centered drug development.
- MSA remains a rare, rapidly progressing neurodegenerative disease with no approved therapies and significant unmet medical need.last yearAmlenetug Receives FDA Fast Track Designation for Multiple System Atrophy- The FDA has granted Fast Track Designation to Lundbeck's amlenetug for the potential treatment of Multiple System Atrophy (MSA).
- The designation is supported by Phase II AMULET trial outcomes and will expedite the drug development process through rolling reviews and intensive guidance.
- Amlenetug, a human monoclonal antibody, targets extracellular α-synuclein to prevent uptake and aggregation, potentially slowing MSA progression.
- Lundbeck has initiated the Phase III MASCOT trial to evaluate the efficacy, safety, and tolerability of amlenetug in MSA patients across North America, Europe, and Asia.last year