Trial Outcomes & Findings for Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia (NCT NCT00832000)

NCT ID: NCT00832000

Last Updated: 2013-08-23

Results Overview

Stiffness measured on a 1-9 scale, 1 being minimal, 9 the worst ever experienced. 0=no symptom reported. For analysis the average severity of stiffness for each participant was calculated from daily calls made in weeks 3-4 of each period.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 59 participants
• Primary outcome timeframe: Weeks 3-4 of each period
• Results posted on: 2013-08-23

Participant Flow

Eligible participants were at least 16 years of age, had clinical symptoms or signs of NDM, and myotonic potentials on electromyography. Participants were either enrolled in the CINCH NDM Natural History Study, or a new patient with genetically confirmed NDM, or with clinical features of NDM but negative myotonic dystrophy DNA testing.

Patients taking anti-myotonic agents were required to discontinue medications for a wash-out period equal to 7 times the half-life of elimination prior to their baseline visit. Participants were ineligible if they has specific contraindications to taking mexiletine (cardiac conduction defects, hepatic or renal disease, or heart failure).

Participant milestones

Measure	Mexiletine Then Placebo 29 Participants will receive mexiletine for 4 weeks, then no intervention for 1 week, and finally placebo for 4 weeks. Included in anaysis\*: 28 patients \*Modified intention to treat analysis. 1 subject in each group not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period	Placebo Then Mexiletine 30 Participants will receive placebo for 4 weeks, then no intervention for 1 week, and finally mexiletine for 4 weeks. Included in analysis\* 29 patients \*Modified intention to treat analysis. 1 subject in each not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period.
Overall Study STARTED	29	30
Overall Study Crossed Over to Opposite Intervention	25	29
Overall Study COMPLETED	23	29
Overall Study NOT COMPLETED	6	1

Reasons for withdrawal

Measure	Mexiletine Then Placebo 29 Participants will receive mexiletine for 4 weeks, then no intervention for 1 week, and finally placebo for 4 weeks. Included in anaysis\*: 28 patients \*Modified intention to treat analysis. 1 subject in each group not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period	Placebo Then Mexiletine 30 Participants will receive placebo for 4 weeks, then no intervention for 1 week, and finally mexiletine for 4 weeks. Included in analysis\* 29 patients \*Modified intention to treat analysis. 1 subject in each not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period.
Overall Study Adverse Event	2	0
Overall Study Withdrawal by Subject	1	0
Overall Study No calls to IVR in either period	1	1
Overall Study No calls to IVR in period 2	2	0

Baseline Characteristics

Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia

Baseline characteristics by cohort

Measure	All Study Participants n=59 Participants All participants received all inerventions; therefore, we combined all participants into one Arm/Group.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	56 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age Continuous	42.9 years STANDARD_DEVIATION 25 • n=5 Participants
Sex: Female, Male Female	26 Participants n=5 Participants
Sex: Female, Male Male	33 Participants n=5 Participants
Region of Enrollment United States	31 participants n=5 Participants
Region of Enrollment Canada	4 participants n=5 Participants
Region of Enrollment United Kingdom	12 participants n=5 Participants
Region of Enrollment Italy	12 participants n=5 Participants

PRIMARY outcome

Timeframe: Weeks 3-4 of each period

Population: Modified intention to treat analysis (n=57). 2 subjects were excluded from analysis due to failure call the IVR system in either period. Treatment group estimates by period are taken from the mixed model, the number above reflecting the number who contributed to the model point estimate. Confidence intervals are bootstrap confidence intervals.

Stiffness measured on a 1-9 scale, 1 being minimal, 9 the worst ever experienced. 0=no symptom reported. For analysis the average severity of stiffness for each participant was calculated from daily calls made in weeks 3-4 of each period.

Outcome measures

Measure	Mexiletine - Period 1 n=28 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=29 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 n=29 Participants Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 n=25 Participants Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Patient-reported Stiffness on the IVR	2.53 units on a scale Interval 1.8 to 3.17	4.21 units on a scale Interval 3.4 to 5.2	1.60 units on a scale Interval 1.04 to 2.2	5.27 units on a scale Interval 4.44 to 6.27

SECONDARY outcome

Timeframe: Weeeks 3-4 of each period

Population: 48 partipants who experienced pain in either period 1 or period 2 were included in analysis. All treatment group means are extracted from the mixed effects model. Confidence intervals are bootstrap confidence intervals.

Pain measured on a 1-9 scale, 1 being minimal, 9 the worst ever experienced. 0=no symptom reported. For analysis the average severity of pain for each participant was calculated from daily calls made in weeks 3-4 of each period.

Outcome measures

Measure	Mexiletine - Period 1 n=48 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=48 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Patient Reported Pain on the IVR	1.54 units on a scale Interval 0.924 to 2.13	3.17 units on a scale Interval 2.43 to 3.93	—	—

SECONDARY outcome

Timeframe: Weeks 3-4 of each period

Population: 44 partipants who experienced weakness in either period 1 or period 2 were included in analysis. All treatment group means are extracted from the mixed effects model. Confidence intervals are bootstrap confidence intervals.

Weakness measured on a 1-9 scale, 1 being minimal, 9 the worst ever experienced. 0=no symptom reported. For analysis the average severity of weakness for each participant was calculated from daily calls made in weeks 3-4 of each period.

Outcome measures

Measure	Mexiletine - Period 1 n=44 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=44 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Patient Reported Weakness on the IVR	1.96 units on a scale Interval 1.42 to 2.63	3.22 units on a scale Interval 2.52 to 3.98	—	—

SECONDARY outcome

Timeframe: Weeks 3-4 of each period

Population: 49 partipants who experienced tiredness in either period 1 or period 2 were included in analysis. All treatment group means are extracted from the mixed effects model. Confidence intervals are bootstrap confidence intervals.

Tiredness measured on a 1-9 scale, 1 being minimal, 9 the worst ever experienced. 0=no symptom reported. For analysis the average severity of tiredness for each participant was calculated from daily calls made in weeks 3-4 of each period.

Outcome measures

Measure	Mexiletine - Period 1 n=49 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=49 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Patient Reported Tiredness on the IVR	2.90 units on a scale Interval 2.12 to 3.68	3.82 units on a scale Interval 3.03 to 4.53	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with quantitative handgrip myotonia values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is a geometric-like mean using log (t+0.1) 'normalizing' transformation. Confidence intervals are bootstrap confidence intervals.

Maximum voluntary contractions following forced right hand grip were recorded and the time to relax from 90% to 5% of average maximal force was determined using automated analysis software.

Outcome measures

Measure	Mexiletine - Period 1 n=54 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=54 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Quantitative Measure of Hand Grip Myotonia (Seconds)	0.321 seconds Interval 0.274 to 0.37	0.429 seconds Interval 0.365 to 0.517	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with short exercise test values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

The maximal post-exercise compound muscle action potential (CMAP) after short periods of exercise as a percent of the baseline measurement.

Outcome measures

Measure	Mexiletine - Period 1 n=56 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=56 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Compound Motor Action Potentials After Short Exercise Test	83.1 percentage of baseline CMAP amplitude Interval 77.5 to 88.4	78.6 percentage of baseline CMAP amplitude Interval 71.9 to 84.7	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with graded needle EMG of the RADM values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

Measured the amount of myotonia present on needle exam by assigning a number 1-3, with 1 being minimal amount of myotonia on needle stick and 3 being maximal amount of myotonia present on needle stick.

Outcome measures

Measure	Mexiletine - Period 1 n=56 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=56 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Graded Myotonia by Needle Electromyography - Right Abductor Digiti Minimi	2.05 units on a scale Interval 1.75 to 2.33	2.62 units on a scale Interval 2.39 to 2.86	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and the end of period 2 (week 9)

Population: All participants with clinical handgrip myotonia values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is a geometric-like mean. Confidence intervals are bootstrap confidence intervals.

The time to open the fist after a forced handgrip as measured on a stopwatch.

Outcome measures

Measure	Mexiletine - Period 1 n=57 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=57 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Clinical Hand Grip Myotonia Evaluation (Seconds)	0.164 Seconds Interval 0.0858 to 0.294	0.494 Seconds Interval 0.281 to 0.872	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and the end of period 2 (week 9)

Population: All participants with clinical eye closure myotonia values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is a geometric-like mean. Confidence intervals are bootstrap confidence intervals.

Time to open the eyes after forced eye closure as measured on a stopwatch.

Outcome measures

Measure	Mexiletine - Period 1 n=57 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=57 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Clinical Eye Closure Myotonia Evaluation (Seconds)	0.161 Seconds Interval 0.0704 to 0.314	0.474 Seconds Interval 0.261 to 0.871	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with graded needle EMG of the RTA values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

Measured the amount of myotonia present on needle exam by assigning a number 1-3, with 1 being minimal amount of myotonia on needle stick and 3 being maximal amount of myotonia present on needle stick.

Outcome measures

Measure	Mexiletine - Period 1 n=56 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=56 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Graded Myotonia by Needle Electromyography - Right Tibialis Anterior	2.07 units on a scale Interval 1.73 to 2.37	2.54 units on a scale Interval 2.28 to 2.76	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with long exercise test values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

Compound muscle action potential (CMAP) after long periods of exercise as a percentage of baseline.

Outcome measures

Measure	Mexiletine - Period 1 n=56 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=56 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Compound Motor Action Potentials After Long Exercise Test	81.8 percentage of baseline CMAP amplitude Interval 76.8 to 87.0	80.1 percentage of baseline CMAP amplitude Interval 74.7 to 86.4	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: All participants with INQoL summary score values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

Quality of life scale for patinets with neuromuscular disorders. The INQoL summary score is a weighted average made up of 5 subdomains (activities, social relationships, independence, emotions, and body image) which document the impact of a disease on a patients' quality of life. Scores range from 0-100, and can be interpreted as the percent of maximal detrimental impact on quality of life. A higher score indicates more detrimental impact.

Outcome measures

Measure	Mexiletine - Period 1 n=51 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=51 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Individualized Neuromuscular Quality of Life Scale - Summary Score	14.0 units on a scale Interval 11.6 to 16.5	16.7 units on a scale Interval 14.0 to 19.4	—	—

SECONDARY outcome

Timeframe: Particiapnts who experienced weakness on mexiletine in either period 1 or period 2.

Population: All participants with SF-36 physical composite values in either period 1 or period 2 were included in analysis. The treatment-specific group mean is taken from the mixed model. Confidence intervals are bootstrap confidence intervals.

The SF-36 is a standard quality of life instrument. The physical composite score represents the the physical burden on quality of life and is a summary of questions related to physical impact of a disease or condition (physical function, role physical, bodily pain, and general health). The score is nomralized to the population and ranges from 0-100, with the US normal value of 50. A lower score represents a greater impact of quality of life.

Outcome measures

Measure	Mexiletine - Period 1 n=57 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=57 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Short Form 36 - Physical Composite Score	44.8 units on a scale Interval 41.9 to 47.4	39.2 units on a scale Interval 35.9 to 41.9	—	—

SECONDARY outcome

Timeframe: The end of period 1 (week 4) and period 2 (week 9)

Population: Modified intention to treat analysis (n=57). 2 subjects were excluded from analysis due to failure call the IVR system in either period. Treatment group estimates by period are taken from the mixed model, the number above reflecting the number who contributed to the model point estimate. Confidence intervals are bootstrap confidence intervals.

The SF-36 is a standard quality of life instrument. The mental composite score represents the the mental burden on quality of life and is a summary of questions related to mental impact of a disease or condition (mental function, role emotional, vitality, and mental health). The score is nomralized to the population and ranges from 0-100, with the US normal value of 50. A lower score represents a greater impact of quality of life.

Outcome measures

Measure	Mexiletine - Period 1 n=28 Participants Mexiletine capsules 200 mg orally three times daily period 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 1 n=29 Participants Placebo capsules orally three times dailyperiod 1. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Mexiletine - Period 2 n=29 Participants Mexiletine capsules 200 mg orally three times daily period 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.	Placebo - Period 2 n=25 Participants Placebo capsules orally three times dailyperiod 2. Due to an interaction between period and treatment the primary outcome was presented separately for periods 1 and 2.
Short Form 36 - Mental Composite Score	47.4 units on a scale Interval 44.0 to 50.2	47.7 units on a scale Interval 44.2 to 51.3	53.1 units on a scale Interval 50.3 to 55.8	42.7 units on a scale Interval 36.8 to 48.3

Adverse Events

Mexiletine Treatment

Serious events: 1 serious events

Other events: 24 other events

Deaths: 0 deaths

Placebo Treatment

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	Mexiletine Treatment n=57 participants at risk Adverse events that occurred when patients were taking placebo.	Placebo Treatment n=57 participants at risk Adverse events that occurred when patients were taking mexiletine.
Psychiatric disorders Drug withdrawal	100.0% 1/1 • Number of events 1 • Data was collected over a 3 year period	— 0/0 • Data was collected over a 3 year period

Other adverse events

Measure	Mexiletine Treatment n=57 participants at risk Adverse events that occurred when patients were taking placebo.	Placebo Treatment n=57 participants at risk Adverse events that occurred when patients were taking mexiletine.
General disorders Constitutional	5.3% 3/57 • Number of events 3 • Data was collected over a 3 year period	0.00% 0/57 • Data was collected over a 3 year period
Skin and subcutaneous tissue disorders Dermatologic/skin	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period	3.5% 2/57 • Number of events 2 • Data was collected over a 3 year period
Gastrointestinal disorders Gastrointestinal	15.8% 9/57 • Number of events 9 • Data was collected over a 3 year period	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period
Infections and infestations Infection	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period	5.3% 3/57 • Number of events 3 • Data was collected over a 3 year period
Blood and lymphatic system disorders Lymphatics	0.00% 0/57 • Data was collected over a 3 year period	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period
Musculoskeletal and connective tissue disorders Musculosketetal/soft tissue	0.00% 0/57 • Data was collected over a 3 year period	3.5% 2/57 • Number of events 2 • Data was collected over a 3 year period
Nervous system disorders Neurologic	8.8% 5/57 • Number of events 5 • Data was collected over a 3 year period	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period
General disorders Pain	7.0% 4/57 • Number of events 4 • Data was collected over a 3 year period	0.00% 0/57 • Data was collected over a 3 year period
Cardiac disorders Cardiac	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period	1.8% 1/57 • Number of events 1 • Data was collected over a 3 year period

Additional Information

Richard J. Barohn, MD

University of Kansas Medical Center

Phone: 913-588-6095

Email: rbarohn@kumc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place