Trial Outcomes & Findings for Mexiletine for the Treatment of Muscle Cramps in ALS (NCT NCT01811355)
NCT ID: NCT01811355
Last Updated: 2017-08-28
Results Overview
The average of the daily recording of number of muscle cramps that occurred in the last 24 hours- over a 6 week period.
COMPLETED
PHASE4
23 participants
6 weeks
2017-08-28
Participant Flow
Of 28 patients who signed consent, 5 did not meet inclusion criteria because of past myocardial infarction (n=1), long QT syndrome (n=1), AV block (n=1), no muscle cramps (n=1), and hospitalization during the screening period that resulted in inability to complete a screening diary and follow-up (n=1) .
Participant milestones
| Measure |
Mexiletine First/Placebo Second
Mexiletine, capsule, 150mg, PO BID, 14 days
Placebo, capsule, PO BID, 14 days
|
Placebo First/Mexiletine Second
Placebo, capsule, PO BID, 14 days
Mexiletine, capsule, 150mg, PO BID, 14 days
|
|---|---|---|
|
First Dose
STARTED
|
11
|
12
|
|
First Dose
COMPLETED
|
9
|
12
|
|
First Dose
NOT COMPLETED
|
2
|
0
|
|
Washout
STARTED
|
9
|
12
|
|
Washout
COMPLETED
|
9
|
12
|
|
Washout
NOT COMPLETED
|
0
|
0
|
|
Second Dose
STARTED
|
9
|
12
|
|
Second Dose
COMPLETED
|
9
|
12
|
|
Second Dose
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Mexiletine for the Treatment of Muscle Cramps in ALS
Baseline characteristics by cohort
| Measure |
Total Subjects Enrolled
n=23 Participants
A total of 23 subjects were enrolled. 11 were randomized to receive study drug first, followed by placebo. 12 subjects received placebo first and study drug second. Overall participant characteristics are displayed in the baseline table.
|
|---|---|
|
Age, Customized
Years
|
62.4 years
STANDARD_DEVIATION 12.5 • n=5 Participants
|
|
Sex/Gender, Customized
males
|
15 Participants
n=5 Participants
|
|
Sex/Gender, Customized
females
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 weeksPopulation: Among the 21 patients who completed the study, 1 patient did not return the outcome diary, which resulted in 20 patients available for analysis.
The average of the daily recording of number of muscle cramps that occurred in the last 24 hours- over a 6 week period.
Outcome measures
| Measure |
Mexiletine First/Placebo Second
n=8 Participants
Mexiletine, capsule, 150mg, PO BID, 14 days
Placebo, capsule, PO BID, 14 days
|
Placebo First/Mexiletine Second
n=12 Participants
Placebo, capsule, PO BID, 14 days
Mexiletine, capsule, 150mg, PO BID, 14 days
|
|---|---|---|
|
Daily Muscle Cramps
Placebo Dose
|
6.1 Cramps per 24 hours
Standard Deviation 8.1
|
4.7 Cramps per 24 hours
Standard Deviation 4.2
|
|
Daily Muscle Cramps
Mexiletine Dose
|
4.9 Cramps per 24 hours
Standard Deviation 8.0
|
2.5 Cramps per 24 hours
Standard Deviation 2.8
|
|
Daily Muscle Cramps
Difference Between
|
1.2 Cramps per 24 hours
Standard Deviation 2.9
|
2.2 Cramps per 24 hours
Standard Deviation 2.2
|
PRIMARY outcome
Timeframe: 6 weeksPopulation: Among the 21 patients who completed the study, 1 patient did not return the outcome diary, which resulted in 20 patients available for analysis.
Daily cramp severity was rated on the 100-unit visual analog scale. Scores ranged from 0 to 100, with 100 being the greatest amount of cramp severity
Outcome measures
| Measure |
Mexiletine First/Placebo Second
n=8 Participants
Mexiletine, capsule, 150mg, PO BID, 14 days
Placebo, capsule, PO BID, 14 days
|
Placebo First/Mexiletine Second
n=12 Participants
Placebo, capsule, PO BID, 14 days
Mexiletine, capsule, 150mg, PO BID, 14 days
|
|---|---|---|
|
Cramp Severity
Placebo Dose
|
43.6 units on a scale
Standard Deviation 31.0
|
44.7 units on a scale
Standard Deviation 26.7
|
|
Cramp Severity
Mexiletine Dose
|
23.1 units on a scale
Standard Deviation 16.8
|
32.8 units on a scale
Standard Deviation 27.7
|
|
Cramp Severity
Difference Between
|
20.5 units on a scale
Standard Deviation 40.6
|
12.0 units on a scale
Standard Deviation 18.1
|
Adverse Events
Mexiletine
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Mexiletine
n=23 participants at risk
Mexiletine, capsule, 150mg, PO BID, 14 days
Mexiletine: Sodium channel blocker
|
Placebo
n=21 participants at risk
Placebo, capsule, PO BID, 14 days
Placebo: Placebo
|
|---|---|---|
|
Nervous system disorders
Dizzines
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Nervous system disorders
Fall
|
4.3%
1/23 • Number of events 1
|
4.8%
1/21 • Number of events 1
|
|
Nervous system disorders
Headache
|
4.3%
1/23 • Number of events 1
|
4.8%
1/21 • Number of events 1
|
|
Ear and labyrinth disorders
Congestion
|
0.00%
0/23
|
4.8%
1/21 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Chest tightness
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Gastrointestinal disorders
Dry mouth
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Renal and urinary disorders
Increased urination
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Skin and subcutaneous tissue disorders
Itchiness
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Gastrointestinal disorders
Nausea
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
|
Gastrointestinal disorders
Diarrhea
|
4.3%
1/23 • Number of events 1
|
0.00%
0/21
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place