Trial Outcomes & Findings for Modafinil for Treatment of Fatigue in ALS Patients (NCT NCT00614926)

Last Updated: 2012-02-20

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

32 participants

Primary outcome timeframe

4 weeks

Results posted on

2012-02-20

Participant Flow

dates of recruitment were June 2006 through October 2007 at at the MDA-ALS Neurology Clinic at Columbia University.

Medical exclusion criteria were low thyroid levels which led to 3 patients excluded.

Participant milestones

Participant milestones
Measure	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.	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.
Overall Study STARTED	25	7
Overall Study COMPLETED	21	7
Overall Study NOT COMPLETED	4	0

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	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.
Overall Study Burden of travel	1	0
Overall Study Adverse Event	3	0

Baseline Characteristics

Modafinil for Treatment of Fatigue in ALS Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Modafinil n=25 Participants 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.	Placebo n=7 Participants 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.	Total n=32 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	23 Participants n=5 Participants	6 Participants n=7 Participants	29 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age Continuous	59 years STANDARD_DEVIATION 13 • n=5 Participants	56 years STANDARD_DEVIATION 5 • n=7 Participants	58 years STANDARD_DEVIATION 12 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants	4 Participants n=7 Participants	18 Participants n=5 Participants
Region of Enrollment United States	25 participants n=5 Participants	7 participants n=7 Participants	32 participants n=5 Participants
Fatigue Severity Scale score >40	25 participants n=5 Participants	7 participants n=7 Participants	32 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

Population: 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.

Outcome measures

Outcome measures
Measure	Modafinil n=25 Participants 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.	Placebo n=7 Participants 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.
Participants Considered "Responders" (Scored 1 or 2) on Clinical Global Impressions Scale	19 participants considered "responders"	1 participants considered "responders"

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

Adverse Events

Modafinil

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Judith Rabkin, PhD

Columbia University

Phone: 212 543 5762

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place