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The purpose of this pilot study is to evaluate whether modafinil is helpful in alleviating fatigue, low energy, drowsiness and difficulty concentrating among patients with amyotrophic lateral sclerosis (ALS), and to evaluate incidence and frequency of adverse events, if any.
ALS is an untreatable, progressive, fatal neurodegenerative disease whose etiology is unknown and whose course is relatively rapid (median survival 3 years after diagnosis). Palliative care, including symptom management, can contribute greatly to improved quality of life. In this context, alleviation of fatigue can help maintain function, extend the duration of time when employment is feasible for those still working, and can enable patients to more fully participate in and enjoy social and recreational activities. Given the prevalence of fatigue in this population, identification of effective treatment is a meaningful goal.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Modafinil | Experimental | Eligible patients will be treated at baseline through Week 4. Those who choose to continue will have additional in-person visits at Weeks 8 and 12 visits (and Week 16 for those starting modafinil at Week 4). |
|
| Placebo | Placebo Comparator | Sugar pill equivalent to the active comparator. Dosing schedule will be the same as the dosing schedule for Modafinil. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Modafinil | Drug | Dose schedule: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day as clinically indicated, in the absence of dose-limiting side effects. Dose is daily, in A.M., for 4 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Participants Considered "Responders" (Scored 1 or 2) on Clinical Global Impressions Scale | The CGI is a standardized assessment tool widely used in clinical psychopharmacology trials as an outcome measure. Scores range from 1= very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5-7 = worse. We use it as a dichotomous measure with scores of 1 or 2 signifying "responder" and all the rest as "non-responder" using all available data including clinician judgement, and ratings scales. | 4 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Number of "Impaired" Scores on Neuropsychological (Brief) Test Battery | This was an initial plan but the large majority of patients were too impaired (either anarthric or unable to use hands) to complete the tests we had selected so this outcome measure turned out to be unfeasible. Therefore, 0 participants were analyzed. | 4 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Judith G Rabkin, PhD | professor | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| New York State Psychiatric Institute-Columbia University | New York | New York | 10032 | United States |
Medical exclusion criteria were low thyroid levels which led to 3 patients excluded.
dates of recruitment were June 2006 through October 2007 at at the MDA-ALS Neurology Clinic at Columbia University.
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| ID | Title | Description |
|---|---|---|
| FG000 | Modafinil | Dose schedule: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day as clinically indicated, in the absence of dose-limiting side effects. Dose is daily, in A.M., for 4 weeks. |
| FG001 | Placebo | Placebo capsules are administered on the same schedule as active drug: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day in the absence of clinical improvement and dose limiting side effects. Dose is daily, in A.M. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Modafinil | Dose schedule: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day as clinically indicated, in the absence of dose-limiting side effects. Dose is daily, in A.M., for 4 weeks. |
| BG001 | Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Participants Considered "Responders" (Scored 1 or 2) on Clinical Global Impressions Scale | The CGI is a standardized assessment tool widely used in clinical psychopharmacology trials as an outcome measure. Scores range from 1= very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5-7 = worse. We use it as a dichotomous measure with scores of 1 or 2 signifying "responder" and all the rest as "non-responder" using all available data including clinician judgement, and ratings scales. | Analysis was Intention to Treat (ITT) including Last-Observation-Carried-Forward (LOCF) as indicated. Proof of concept study so N was based on accrual feasibility. | Posted | Jun 2010 | Number | participants considered "responders" | 4 weeks |
|
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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 | Modafinil | Dose schedule: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day as clinically indicated, in the absence of dose-limiting side effects. Dose is daily, in A.M., for 4 weeks. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Judith Rabkin, PhD | Columbia University | 212 543 5762 | jgr1@columbia.edu |
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| ID | Term |
|---|---|
| D005221 | Fatigue |
| ID | Term |
|---|---|
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D000077408 | Modafinil |
| D000073893 | Sugars |
| ID | Term |
|---|---|
| D001559 | Benzhydryl Compounds |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
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|
| Placebo | Drug | Placebo capsules are administered on the same schedule as active drug: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day in the absence of clinical improvement and dose limiting side effects. Dose is daily, in A.M. |
|
|
Placebo capsules are administered on the same schedule as active drug: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day in the absence of clinical improvement and dose limiting side effects. Dose is daily, in A.M. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Fatigue Severity Scale score >40 | Minimum score = 9, maximum = 63. Nine items, no subscales. Higher scores signify greater fatigue. Total summed scores over 40 (equal to mean item score of 4.5+) are considered to signify "clinically significant fatigue" and was a requirement for study eligibility. | Number | participants |
|
| OG001 | Placebo | Placebo capsules are administered on the same schedule as active drug: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day in the absence of clinical improvement and dose limiting side effects. Dose is daily, in A.M. |
|
|
| Secondary | Number of "Impaired" Scores on Neuropsychological (Brief) Test Battery | This was an initial plan but the large majority of patients were too impaired (either anarthric or unable to use hands) to complete the tests we had selected so this outcome measure turned out to be unfeasible. Therefore, 0 participants were analyzed. | Posted | Number | participants | 4 weeks |
|
|
| 0 |
| 25 |
| 0 |
| 25 |
| EG001 | Placebo | Placebo capsules are administered on the same schedule as active drug: 50 mg/day for 1 week, increasing to 100 mg/day at Week 2. Thereafter, dose may be increased to 300 mg/day in the absence of clinical improvement and dose limiting side effects. Dose is daily, in A.M. | 0 | 7 | 0 | 7 |
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| D006838 |
| Hydrocarbons |
| D009930 | Organic Chemicals |
| D002241 | Carbohydrates |