Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
|ClinicalTrials.gov Identifier: NCT01406873|
Recruitment Status : Completed
First Posted : August 1, 2011
Results First Posted : March 29, 2018
Last Update Posted : March 29, 2018
|Condition or disease||Intervention/treatment||Phase|
|Myotonic Dystrophy||Drug: Mexiletine Drug: Placebo||Phase 2|
This study will provide data on the long term (6 months) safety and efficacy of mexiletine in:
- improving the distance participants are able to walk in six minutes;
- reducing myotonia;
- improving muscle strength;
- increasing lean muscle mass;
- decreasing musculoskeletal pain;
- improving gastrointestinal function and swallowing);
- improving functional abilities;
- decreasing cardiac arrhythmias; and
- improving disease-specific health related quality-of-life.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||42 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)|
|Study Start Date :||June 2011|
|Actual Primary Completion Date :||February 2017|
|Actual Study Completion Date :||March 2017|
Active Comparator: Mexiletine
20 subjects will be randomized (assigned) to receive Mexiletine. Mexiletine is available on the market for the treatment of cardiac arrhythmias, but it is not currently approved for the treatment of myotonia or myotonic dystrophy.
150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
Other Name: Generic name: mexiletine hydrochloride
Placebo Comparator: Sugar pill
20 subjects will be randomized (assigned) to receive placebo (sugar pill). This control group is necessary to definitely establish the antimyotonic efficacy and safety of mexiletine.
150 mg/kg placebo capsules taken by mouth, three times daily for 6 months
- Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance [ Time Frame: Baseline to 6 months ]During this assessment, participants were asked to walk as far as they could back and forth on a fixed 20 meter route for 6 minutes. The total distance walked during the 6 minutes was recorded in meters. Change from baseline was defined as the difference between the average 6 minute walk distance at baseline and the average 6 minute walk distance at 6 months.
- Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months [ Time Frame: 6 months ]Adverse events were monitored at the three in-person evaluations (Months 0, 3, and 6), at telephone evaluations every 2 weeks, and via patient-completed side effect diaries. The study investigators and safety monitoring committee reviewed adverse events and made decisions regarding drug withdrawals, suspensions, and dose reductions as needed.
- Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia [ Time Frame: Baseline to 6 months ]Relaxation time of the long finger flexor muscles of the right hand after a maximum voluntary isometric contraction performed in a standardized fixed position of the right arm elbow/wrist/hand. Relaxation time for this measurement is defined as the time to relax from 90% to 5% of the maximum isometric force of contraction of the hand (the first of 6 serial contractions averaged over two consecutive trials performed 10 minutes apart).
- Mean Change From Baseline in Manual Muscle Testing (MMT) Score [ Time Frame: Baseline to 6 months ]Manual muscle testing was performed on 26 muscle groups (shoulder abductors, elbow flexors, wrist flexors, wrist extensors, hip flexors, knee extensors, hip extensors, knee flexors, hip abductors, elbow extensors, ankle dorsiflexors, and plantar flexors on the right and left plus neck extensor and neck flexors). The muscles were tested in various positions including sitting, supine, prone, and side lying and each graded on a modification of the Medical Research Council (MRC) scale of 0 to 5 (5 representing normal strength).
- Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring [ Time Frame: Baseline to 6 Months ]PR, QRS, and QTc intervals as well as average minimum heart rate (HR) were obtained through standard 12 lead electrocardiograms (ECGs). Values were computer generated and verified by the study investigator and study cardiologist.
- Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life [ Time Frame: Baseline to 6 months ]
- The Myotonic Dystrophy Health Index (MDHI) is a validated disease-specific measure of patient-reported disease burden. The MDHI total score is a weighted average derived from 17 subscales. MDHI total scores range form 0-100 with 0 representing no patient-reported disease burden and 100 representing the most severe patient-reported disease burden.
- The Individualized Neuromuscular Quality of Life Questionnaire (INQoL) is a measure of quality of life in neuromuscular disease. The INQoL summary score is a weighted average made up of 5 sub-domains. Scores range from 0-100, and can be interpreted as the percent of maximal detrimental impact on quality of life with higher scores indicating more detrimental impact.
- The 36-Item Short Form Survey (SF-36) is a generic measure of quality of life across 8 domains. Two summary metrics are produced from the 8 domains, ranging from 0-100% with lower scores representing worse levels of functioning.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01406873
|United States, New York|
|University of Rochester Medical Center, Department of Neurology|
|Rochester, New York, United States, 14642|
|Principal Investigator:||Richard T. Moxley, III, MD||University of Rochester|