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| Name | Class |
|---|---|
| ALS Association | OTHER |
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Trials evaluating new therapies for stopping or slowing the progression of ALS depend critically upon the use of outcome measures to assess whether a potential treatment is effective. The more effective an outcome measure, the fewer patients need to be enrolled and the shorter the trial. Many outcome measures have been used over the years, including strength assessments, breathing tests, functional status surveys, and nerve testing, but all are far from ideal. A new method, called electrical impedance myography (EIM) appears to be especially promising in that it provides very consistent data from one testing session to the next, is sensitive to the muscle deterioration that occurs in ALS, and is entirely painless and non-invasive. In this study, investigators from multiple institutions plan to compare several different outcome measures, including EIM, in approximately 120 ALS patients, with each patient being followed for a period of one year. All of these measures will be compared to one another and an assessment of their ability to detect disease progression made. Our goal will be to determine whether EIM can serve as a valuable new outcome measure, ultimately leading to substantially faster, more effective ALS trials requiring fewer patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALS patients | Patients with clinically established amyotrophic lateral sclerosis |
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| Measure | Description | Time Frame |
|---|---|---|
| Electrical Impedance Myography | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| ALS Functional Rating Scale | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with amyotrophic lateral sclerosis (ALS)
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| Name | Affiliation | Role |
|---|---|---|
| Seward B Rutkove, MD | Beth Israel Deaconess Medical Center | Principal Investigator |
| Jeremy M Shefner, MD, PhD | Upstate Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States | ||
| Emory University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 17897874 | Background | Rutkove SB, Zhang H, Schoenfeld DA, Raynor EM, Shefner JM, Cudkowicz ME, Chin AB, Aaron R, Shiffman CA. Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials. Clin Neurophysiol. 2007 Nov;118(11):2413-8. doi: 10.1016/j.clinph.2007.08.004. Epub 2007 Sep 25. |
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| ID | Title | Description |
|---|---|---|
| FG000 | ALS Patients | Patients with clinically established amyotrophic lateral sclerosis |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 6 months |
| Handheld Dynamometry | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. | 6 months |
| Atlanta |
| Georgia |
| United States |
| Johns Hopkins | Baltimore | Maryland | United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02446 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | United States |
| Upstate Medical Center | Syracuse | New York | United States |
| Wake Forest University Baptist Medical Center | Winston-Salem | North Carolina | 27157 | United States |
| University of Virginia Medical Center | Charlottesville | Virginia | 22908 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | ALS Patients | Patients with clinically established amyotrophic lateral sclerosis |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Electrical Impedance Myography | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. | Posted | Mean | 95% Confidence Interval | Coefficient of Variation | 6 months |
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| Secondary | ALS Functional Rating Scale | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. | Posted | Mean | 95% Confidence Interval | Coefficient of variation | 6 months |
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| Secondary | Handheld Dynamometry | The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure. | Posted | Mean | 95% Confidence Interval | Coefficient of variation | 6 months |
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ALS Patients | Patients with clinically established amyotrophic lateral sclerosis | 0 | 89 | 0 | 89 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Seward Rutkove | Beth Israel Deaconess Medical Center | 617-667-8130 | srutkove@bidmc.harvard.edu |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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