Exploratory Study of Biotelemetry in Amyotrophic Lateral Sclerosis
2018年5月31日 更新者：GlaxoSmithKline
An Exploratory Study to Investigate the Use of Biotelemetry to Identify Markers of Disease Progression in Subjects With Amyotrophic Lateral Sclerosis
Study 201283 is an exploratory, non-controlled, non-drug study in Amyotrophic Lateral Sclerosis (ALS) subjects. This study is being conducted as the first step for developing new meaningful measure(s) which might prove to be more effective than existing measures for monitoring clinical function and disease course in ALS. The objective of this study is to test novel measures of movement/physical activity, heart rate and speech and explore how they measure disease progression by evaluating their relationship to gold standard measures of function. This study will be conducted in two phases. A variable length Pilot Phase to test biotelemetry instruments and algorithms reliability and ease of use/acceptance. Approximately 5 subjects will have at least 1 clinic visit to perform a series of set reference tasks while wearing the accelerometer and electrode. Subjects will also continuously wear the accelerometer and electrode in their routine home-life setting for approximately 3 days after the clinic visit (i.e., home monitoring). Subjects in the Pilot Phase will continue in the study and participate in the Core Study Phase. A 48 week Core Study Phase will be conducted to evaluate how measures of movement/physical activity, speech and Heart Rate Variability (HRV) relate to ALS disease progression. During this phase, a maximum of 25 subjects will be enrolled. Subjects will attend 5 clinic visits to perform gold standard measures of function and perform a series of set reference tasks while wearing the accelerometer and electrode. In between clinic visits, every month subjects will attach the accelerometer and electrode and wear it for approximately 3 days in their home. A telephone contact with the subject will be made by the site at the end of each 3-day home monitoring period.
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研究概览
地位
完全的
条件
研究类型
介入性
注册 (实际的)
25
阶段
- 不适用
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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London、英国、SE5 8AF
- GSK Investigational Site
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Oxford、英国、OX3 9DU
- GSK Investigational Site
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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
18年 至 80年 (成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Between 18 and 80 years of age, inclusive, at the time of signing the informed consent.
- Diagnosed with ALS by a neurologist with expertise in ALS. For subjects with bulbar onset there must be objective limb involvement of at least one limb.
- Diagnosed with ALS within 18 months of symptom onset.
- Subjects must be ambulatory (i.e., must not be confined to a wheelchair).
- Male and female subjects.
- Capable of giving signed (or verbal consent or assent where applicable) informed consent as described in Protocol which includes compliance with the requirements and restrictions listed in the consent form and in protocol.
- Capable and willing to comply with the requirements of the protocol (either by themselves or with assistance).
Exclusion Criteria:
- Neurological (other than the subject's ALS) or non-neurological co-morbidities (e.g. joint disease, respiratory disease) which limit mobility.
- Clinically significant cognitive impairment in the opinion of the investigator.
- Regionally restricted forms of ALS, or other atypical variants: Isolated corticobulbar pattern of ALS with normal ambulation; Flail arm syndrome; Primary lateral sclerosis; Signs of chronic partial denervation restricted to a single limb; ALS parkinsonism dementia complex
- Subjects requiring mechanical ventilation (non-invasive ventilation for sleep apnoea is allowed).
- Historical or current evidence of clinically significant uncontrolled disease which, in the opinion of the investigator, would put the safety of the subject at risk through participation or impact the study assessments or endpoints.
- Presence of an active implantable cardiac medical device (e.g., pacemaker or implantable cardioverter-defibrillator) or at a high risk for needing external defibrillation.
- History of skin hypersensitivity to adhesives.
- Current participation in a clinical trial which in the opinion of the investigator and GSK medical monitor might impact the objectives of this study.
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
- 主要用途：其他
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的：Pilot and Core Study Phase
During Pilot phase,subjects will attend clinic at least once to perform a series of set reference tasks while wearing the accelerometer and electrode.
Subjects will also continuously wear the accelerometer and electrode in their routine home-life setting for approximately 3 days after the clinic visit (home monitoring).
During 48 week Core Study, subjects will attend 5 clinic visits to perform gold standard measures of function (ALS Functional Rating Scale-Revised and Forced Vital Capacity) and perform a series of set reference tasks while wearing the accelerometer and electrode.
Subjects will also continuously wear the accelerometer and electrode in their routine home-life setting for approximately 3 days after the clinic visits (home monitoring).
In between clinic visits, subjects will attach the accelerometer and electrode and wear it for approximately 3 days in their home.
A telephone contact with the subject will be made by the site at the end of each 3-day home monitoring period
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Each subject will be issued one FS and a MAT LifeInsight Hub for use during the study.
At each clinic visit in the Pilot and Core Study Phases, the Faros sensor will be placed on the subject just prior to the defined reference tasks and will be worn on the subject's sternum during completion of the tasks.
The morning after the clinic visit, the subject will re-attach the sensor and wear it for approximately 3 days.
In between visits during the Core Study Phase the subject will wear the FS every month for approximately 3 days to enable data collection on a monthly basis over the 48 week study period.
Movement/physical activity data will be collected by the FS throughout the study.
The Fast Fix electrode patch will be worn with the FS on the subject's sternum according to the same schedule as the FS.
The Fast Fix electrode patch will be replaced by the subject on a daily basis during the 3 day monitoring period.
Subjects will be provided instructions on how to operate and wear the Fast Fix electrode patch.
Subjects will follow simple prompts on a computer screen instructing them to say a series of vowels, words, and paragraphs which will be recorded using a high definition digital microphone and stored securely on a laptop.
The speech waveform data will be sent via secure method to GSK/MAT for processing.
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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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Duration of Day Time Wear Time of the Device
大体时间：Up to Week 48
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Each participant was provided one accelerometer and electrode (Faros sensor and LifeInsight Hub) through which movement/physical activity data was collected throughout the study.
Duration of day time wear time was calculated by adding the durations of the time spent [Active + lying + sedentary not lying] in the day time.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).NA indicates that data could not be calculated as participant number was <=1.
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Up to Week 48
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Duration of Night Time Wear Time of the Device
大体时间：Up to Week 48
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Each participant was provided one accelerometer and electrode (Faros sensor and LifeInsight Hub) through which movement/physical activity data was collected throughout the study.
Duration of night time wear time was the calculated as the total of the times spent [Active + day time lying + day time "sedentary not lying"] for the night time.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that data could not be calculated as participant number was <=1.
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Up to Week 48
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Average Time Spent Active
大体时间：Up to Week 48
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Number of minutes spent active per day and night over the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles)
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Up to Week 48
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Average Time Spent 'Sedentary Not Lying'
大体时间：Up to Week 48
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Number of minutes spent 'sedentary not lying' per day and night for 24-hour recording period; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Average Time Spent Lying
大体时间：Up to Week 48
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Number of minutes spent lying per day and night over the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Average Time Spent Sedentary.
大体时间：Up to Week 48
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Number of minutes spent sedentary [time spent lying + time spent sedentary not lying] per day and night over the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Time Spent With Sensor Off
大体时间：Up to Week 48
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Sensor off time includes the time that the sensor was either switched off or the participant was not wearing it (or both).
This outcome measure was planned but not performed.
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Up to Week 48
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Total Activity Count
大体时间：Up to Week 48
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Total activity count for the day time and night time for the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Maximum Activity Count in a 24 Hour Period
大体时间：Up to Week 48
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Maximum activity count for the day time and night time for the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles)
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Up to Week 48
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Mean Maximum Activity Count in a 24 Hour Period
大体时间：Up to Week 48
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Mean maximum activity count of day time and night time for the 24-hour recording periods; averaged for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles)
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Up to Week 48
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Number of Continuous Active Periods
大体时间：Up to Week 48
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Active periods were catagorized as >1minute to <=2minutes, >2 minutes to <=5minutes, >5 minutes to <=15 minutes, >15 minutes to <=30 minutes, >30 minutes.
Total number of 'active periods were calculated as 1minute<x<2minutes + 'number of active periods 2minutes<x<5minutes + 'number of active periods 5minutes<x<15minutes + 'number of active periods 15minutes<x<30minutes + 'number of active periods >30minutes.
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Up to Week 48
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Percent of Time Lying Down at Night
大体时间：Up to Week 48
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Percent time spent lying per day and night over the 24-hour recording periods; averaged for each timepoint
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Up to Week 48
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Number of Night Time Movement Episodes Per Hour
大体时间：Up to Week 48
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Average number of night movement episodes per hour for each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Percent Time Night-time Rest Efficiency
大体时间：Up to Week 48
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Average night-time rest efficiency for each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles)
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Up to Week 48
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Rest Fragmentation Index
大体时间：Up to Week 48
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Rest Fragmentation Index was computed as movement time (%) divided by number of movement episodes for each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Average Duration of Night Time Movement Episodes
大体时间：Up to Week 48
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Average duration of movement episodes for each time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Relationship Between Average Number Night Time Movements/Hour Versus Total ALSFRS-R
大体时间：Up to Week 48
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Estimates for between and within subject correlation coefficients were produced following multiple linear regression analyses on the actigraphy endpoints, comparing them with the ALSFRS-R total score and the gross motor domain and fine motor domain scores.
Data for between subject correlation coefficient has been presented
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Up to Week 48
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Relationship Between Average Duration Movement Episodes at Night Versus Total ALSFRS-R
大体时间：Up to Week 48
|
Estimates for between and within subject correlation coefficients were produced following multiple linear regression analyses on the actigraphy endpoints, comparing them with the ALSFRS-R total score and the gross motor domain and fine motor domain scores.
Data for between subject correlation coefficient has been presented
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Up to Week 48
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Relationship Between Average Percent Time Night Time Rest Efficiency Versus Total ALSFRS-R
大体时间：Up to Week 48
|
Estimates for between and within subject correlation coefficients were produced following multiple linear regression analyses on the actigraphy endpoints, comparing them with the ALSFRS-R total score and the gross motor domain and fine motor domain scores.
Data for between subject correlation coefficient has been presented
|
Up to Week 48
|
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Relationship Between Average Night Time Rest Fragmentation Index Versus Total ALSFRS-R
大体时间：Up to Week 48
|
Estimates for between and within subject correlation coefficients were produced following multiple linear regression analyses on the actigraphy endpoints, comparing them with the ALSFRS-R total score and the gross motor domain and fine motor domain scores.
Data for between subject correlation coefficient has been presented
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Up to Week 48
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Mean Heart Rate Variability (HRV) While Lying (Low Frequency[LF]/High Frequency[HF])
大体时间：Up to Week 48
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Mean Heart Rate Variability (HRV) averaged over 5 windows of lying down at each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Variance of HRV While Lying (LF/HF)
大体时间：Up to Week 48
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Variance of Heart Rate Variability (HRV) averaged over 5 windows of lying down at each protocol time point (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Mean HRV While Sedentary Not Lying (LF/HF)
大体时间：Up to Week 48
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Mean Heart Rate Variability (HRV) averaged over 5 windows of sedentary not lying at each protocol time point (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Variance of HRV While Sedentary Not Lying (LF/HF)
大体时间：Up to Week 48
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Variance of Heart Rate Variability (HRV) averaged over 5 windows of sedentary not lying at each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Mean HRV While Active (LF/HF)
大体时间：Up to Week 48
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Mean Heart Rate Variability (HRV) averaged over 5 windows of subjects being active at each protocol time point (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that data is not available.
Standard deviation could not be calculated when number of participants was <=1
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Up to Week 48
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Variance of HRV While Active (LF/HF)
大体时间：Up to Week 48
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Variance of Heart Rate Variability (HRV) averaged over 5 windows of subjects being active at each protocol time point (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that data could not be calculated as participant number was <=1
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Up to Week 48
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Effect of Being Upright on HRV- Mean HRV (LF/HF Analysis)
大体时间：Up to Week 48
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The effect of being upright on mean HRV was calculated as [mean HRV while sedentary not lying minus mean HRV while lying] (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Effect of Being Upright on HRV Variance (LF/HF Analysis)
大体时间：Up to Week 48
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The effect of being upright on HRV variance was calculated as [HRV variance while sedentary not lying minus HRV variance while lying] (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Effect of Activity on Mean HRV (LF/HF)
大体时间：Up to Week 48
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The effect of activity on mean HRV was calculated as [mean HRV while sedentary not lying minus mean HRV while lying] (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that data could not be calculated as participant number was <=1.
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Up to Week 48
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Effect of Activity on HRV Variance (LF/HF Analysis)
大体时间：Up to Week 48
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The effect of activity on HRV variance was calculated as [HRV variance while sedentary not lying minus HRV variance while lying] (LF/HF analysis).
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that data could not be calculated as participant number was <=1.
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Up to Week 48
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Mean HRV Over 24 Hours - Mean Root Mean Square of the Successive Differences (RMSSD)
大体时间：Up to Week 48
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Mean HRV over 24 hours (RMSSD analysis), averaged for each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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HRV Variance Over 24 Hours - Mean Root Mean Square of the Successive Differences (RMSSD) Analysis
大体时间：Up to Week 48
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HRV variance over 24 hours (RMSSD analysis), averaged for each protocol time point.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Relationship Between Absolute Values of HRV Endpoints (LF/HF) and Absolute Value of Total ALSFRS-R
大体时间：Up to Week 48
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Estimates for between and within subject correlation coefficients were produced following multiple linear regression analyses on the actigraphy endpoints, comparing them with the ALSFRS-R total score and the gross motor domain and fine motor domain scores.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
NA indicates that sample size was insufficient to calculate correlation coefficient
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Up to Week 48
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Measurement of Speech Quality
大体时间：Up to Week 48
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Speech quality was assessed by Central Tendency of Fundamental Frequency (CTF) F0, jitter, and shimmer for 'short ah' and 'long ah' tests.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Duration of Maximum Gap Between Words
大体时间：Up to Week 48
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Duration of maximum gap between words during running speech to planned to analyze quality of speech however; was not performed.
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Up to Week 48
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Speaking Rate
大体时间：Up to Week 48
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Speaking rate was analyzed during running speech.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Average Phoneme Rate
大体时间：Up to Week 48
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Phoneme rate was analyzed for the single word "doily".
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Maximum Phonation Time
大体时间：Seconds
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Maximum phonation time for the single word "doily" test was analyzed for quality of speech testing.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Seconds
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Percentage Pause Time
大体时间：Up to Week 48
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Percentage pause time for running speech was analyzed.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Relationship Between Absolute Values of Speech Endpoints and Absolute Value of ALSFRS-R
大体时间：Up to Week 48
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Mixed Model was used to calculate Repeated Measures Correlation Coefficients between the two variables when model is converged.
The correlation coefficient among the repeated measurements is same for different variables.
Multiple Linear Regression was used to calculate Within and Between Participant Correlation Coefficients when Mixed Model is not converged.
Data for Repeated Measures Correlation Coefficient has been presented in the table below
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Up to Week 48
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Relationship Between Absolute Values of Speech Endpoints and Absolute Values of FVC
大体时间：Up to Week 48
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Mixed Model was used to calculate Repeated Measures Correlation Coefficients between the two variables when model is converged.
The correlation coefficient among the repeated measurements is same for different variables.
Multiple Linear Regression was used to calculate Within and Between Participant Correlation Coefficients when Mixed Model is not converged.
Data for Between Subject Correlation Coefficient has been presented in the table below
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Up to Week 48
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Number of Participants Reporting Sensor Comfort
大体时间：Up to Week 48
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Participant's feedback on whether the sensor was comfortable to wear was categorized as "yes" and "no".
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants Reporting Impact on Sleep
大体时间：Up to Week 48
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Participant feedback on how much the sensor impacted their sleep was categorized as "not at all", "moderately" and "minimally".
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants With Ease of Setting up and Attaching the Sensor
大体时间：Up to Week 48
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The participants were required to give feedback on how easy was it to set up and/ or attach the sensor and it was categorized as "easy", "neutral" and "difficult".
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants With Corresponding Activity Level Required to Complete Their Job
大体时间：Up to Week 48
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The activity level required by the participant to complete their job was recorded, and was categorized as Not working, Physical activity required, and Sedentary.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants With Corresponding Average Activity Level During Time of Wearing the Sensor
大体时间：Up to Week 48
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Average activity level during the time of wearing the sensor was reported by the participants, and was categorized as very low level activity, low level activity, moderate level activity and high level activity.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants Whose Sensor Fell Off
大体时间：Up to Week 48
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The participants reported whether the sensor fell off.
Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
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Up to Week 48
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Number of Participants With Adverse Events Secondary to the Devices Used or Due to Study Procedures
大体时间：Up to Week 48
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Only those AEs and SAEs which, in the opinion of the investigator, were related to a protocol-mandated procedure or one of the devices used in the study were reported.
An AE is any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Any untoward event resulting in death, life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, congenital anomaly/birth defect, any other situation according to medical or scientific judgment that may not be immediately life-threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention or event associated with liver injury and impaired liver function were categorized as SAE.
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Up to Week 48
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Type of Adverse Events Secondary to the Devices Used or Due to Study Procedures
大体时间：Up to Week 48
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Only those AEs and SAEs which, in the opinion of the investigator, were related to a protocol-mandated procedure or one of the devices used in the study were reported.
An AE is any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Any untoward event resulting in death, life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, congenital anomaly/birth defect, any other situation according to medical or scientific judgment that may not be immediately life-threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention or event associated with liver injury and impaired liver function were categorized as SAE.
Number of AEs, SAEs and Adverse events leading to discontinuation (AELDs)from the study is presented
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Up to Week 48
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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。
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)
2015年6月30日
初级完成 (实际的)
2017年6月1日
研究完成 (实际的)
2017年6月1日
研究注册日期
首次提交
2015年5月11日
首先提交符合 QC 标准的
2015年5月14日
首次发布 (估计)
2015年5月19日
研究记录更新
最后更新发布 (实际的)
2018年12月26日
上次提交的符合 QC 标准的更新
2018年5月31日
最后验证
2017年8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.