Modafinil for Treatment of Fatigue in ALS Patients

Phase 4

Completed

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

Inclusion Criteria

Clinical diagnosis of ALS
Ages 18-80
Clinically significant fatigue (4.5+ on Fatigue Severity Scale with duration 3+ months plus impairment in 1+ categories of role function)
Speaks English
Able and willing to give informed consent
Can communicate verbally or with assistive device
Can swallow capsules
Forced vital capacity 50+%

Exclusion Criteria

Untreated hypothyroidism (TSH > 4.25 UIU/ML)
Untreated and uncontrolled hypertension
Clinically significant anemia (HCT < 33%)
Untreated or under-treated major depressive disorder
Current clinically significant suicidal ideation
Started antidepressant medication for treatment of depression during past 6 weeks
Currently taking psychostimulant medication
History or current psychosis or bipolar disorder
Fecund women not currently using barrier methods of contraception

Arm && Interventions

Group	Intervention	Description
Modafinil	Modafinil	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	Sugar pill equivalent to the active comparator. Dosing schedule will be the same as the dosing schedule for Modafinil.

Primary Outcome Measures

Name	Time	Method
Participants Considered "Responders" (Scored 1 or 2) on Clinical Global Impressions Scale	4 weeks	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.

Secondary Outcome Measures

Name	Time	Method
Number of "Impaired" Scores on Neuropsychological (Brief) Test Battery	4 weeks	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.