Methodology Study of Novel Outcome Measures to Assess Progression of ALS
2019年10月23日 更新者：Biogen
Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of outcome measures administered to participants with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.
研究概览
地位
完全的
条件
研究类型
观察性的
注册 (实际的)
138
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
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Ontario
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Toronto、Ontario、加拿大、M4N 3M5
- Sunnybrook Health Sciences Centre
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Quebec
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Montréal、Quebec、加拿大、H3A 2B4
- Montreal Neurological Institute Clinical Research Unit
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Berlin、德国、13125
- Charite - Campus Virchow-Klinikum
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Hannover、德国、30625
- Medizinische Hochschule Hannover
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Jena、德国、07743
- Universitaetsklinikum Jena
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Ulm、德国、89081
- Universitaetsklinikum Ulm
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Leuven、比利时、3000
- UZ Leuven
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Hérault
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Montpellier、Hérault、法国、34295
- Hopital Gui de Chauliac, Service de Neurologie
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Paris
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Paris cedex 13、Paris、法国、75013
- Groupe Hospitalier Pitie-Salpetriere
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Dublin、爱尔兰、Dublin 9
- Beaumont Hospital
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St. Gallen、瑞士、9007
- Kantonsspital St. Gallen
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California
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San Diego、California、美国、92103
- University of California San Diego Medical Center
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San Francisco、California、美国、94115
- California Pacific Medical Center
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Florida
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Tampa、Florida、美国、33612
- University of South Florida
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Georgia
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Atlanta、Georgia、美国、30322
- The Emory Clinic
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Maryland
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Baltimore、Maryland、美国、21287
- Johns Hopkins Hospital
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Massachusetts
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Charlestown、Massachusetts、美国、2129
- Massachusetts General Hospital, MA
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Missouri
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Saint Louis、Missouri、美国、63110
- Washington University School of Medicine
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Pennsylvania
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Hershey、Pennsylvania、美国、EC037
- Penn State Milton S. Hershey Medical Center
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West Midlands
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Sheffield、West Midlands、英国、S102JF
- Royal Hallamshire Hospital
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CX
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Utrecht、CX、荷兰、3584
- UMC Utrecht
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
16年 至 85年 (孩子、成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
取样方法
概率样本
研究人群
Participants suffering from ALS are recruited by participating physicians in a standard clinical practice setting.
描述
Key Inclusion Criteria:
- A diagnosis of sporadic or familial ALS
- ALS onset within ≤5 years
- Must be 16 to 85 years of age, inclusive, for sites in the United States and 18 to 85 years of age, inclusive, for all sites outside of the United States
Key Exclusion Criteria:
- History of or positive test result at Screening for human immunodeficiency virus (HIV)
- History of or positive test result at Screening for hepatitis C virus (HCV) antibody or hepatitis B virus (HBV)
- Possibility of neuromuscular weakness other than ALS
- Unspecified reasons that, in the opinion of the site Investigator, make the subject unsuitable for enrollment or unlikely to be able to complete, at a minimum, the Month 6 Visit
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
大体时间：Baseline to Month 6 and Baseline to Month 12
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EIM is an electrophysiological technique in which current is applied to a muscle of interest and resultant voltage and impedance are measured.
These measured parameters reflect the conductivity of underlying tissue and presumably the pathologic state of denervated muscle in an ALS participant
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by compound muscle action potential (CMAP)
大体时间：Baseline to Month 6 and Baseline to Month 12
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CMAP is a standard electrophysiological measure generated by maximally stimulating a nerve such that all muscle fibers innervated by the respective nerve are depolarized.
Reduction of CMAP amplitude reflects loss of motor axons and, therefore, is directly relevant to ALS.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number estimation (MUNE)
大体时间：Baseline to Month 6 and Baseline to Month 12
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Optional, to be administered at each site's Investigator's discretion.
MUNE is used to estimate the number of functioning motor units.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number index (MUNIX)
大体时间：Baseline to Month 6 and Baseline to Month 12
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MUNIX estimates functioning motor units within a muscle.
CMAP and surface electromyography potentials (surface interference patterns) are obtained at various levels of voluntary effort, and MUNIX is estimated using power and area of CMAP and surface interference patterns.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in muscle strength measures as assessed by hand-held dynamometry (HHD)
大体时间：Baseline to Month 6 and Baseline to Month 12
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HHD tests isometric strength of multiple muscles using standard participant positioning.
Approximately 10 muscle groups will be examined (per each side) in both upper and lower extremities.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in respiratory measures as assessed by slow vital capacity (SVC)
大体时间：Baseline to Month 6 and Baseline to Month 12
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Vital capacity will be measured by means of an SVC test, administered in the upright position.
Upright SVC will be determined by performing 3 to 5 measures, in accordance with criteria established by the American Thoracic Society and the European Respiratory Society.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in functional measures as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
大体时间：Baseline to Month 6 and Baseline to Month 12
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The ALSFRS-R has been demonstrated to predict survival.
The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills.
There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48 [Cedarbaum 1999], with higher scores representing better function.
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Baseline to Month 6 and Baseline to Month 12
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次要结果测量
结果测量 |
大体时间 |
|---|---|
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Within-participant test-retest reliability between the 2 repeated measurements occurring on Day 1 and Day 7 for EIM
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for CMAP
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for MUNE
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for MUNIX
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for HHD
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for SVC
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for ALSFRS-R
大体时间：Day 1 and Day 7
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Day 1 and Day 7
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Comparison between 6 and 12-month changes in exploratory measures with 18 and 24-month changes in ALSFRS-R and survival
大体时间：Baseline to Month 24
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Baseline to Month 24
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Comparison between 6-month changes for muscle electrophysiological measures
大体时间：Baseline to Month 12
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Baseline to Month 12
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Comparison between 6-month changes for muscle strength measures
大体时间：Baseline to Month 12
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Baseline to Month 12
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Comparison between 6-month changes for functional measures
大体时间：Baseline to Month 12
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Baseline to Month 12
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Comparison of molecular biomarkers with disease progression
大体时间：Baseline to Month 12
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Baseline to Month 12
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
赞助
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2016年1月6日
初级完成 (实际的)
2018年7月27日
研究完成 (实际的)
2019年8月1日
研究注册日期
首次提交
2015年10月8日
首先提交符合 QC 标准的
2015年11月19日
首次发布 (估计)
2015年11月23日
研究记录更新
最后更新发布 (实际的)
2019年10月24日
上次提交的符合 QC 标准的更新
2019年10月23日
最后验证
2019年10月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.