Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)
2 other identifiers
interventional
42
1 country
1
Brief Summary
The purpose of this study is to investigate the effects of mexiletine treatment for 6 months on ambulation, myotonia, muscle function and strength, pain, gastrointestinal functioning, cardiac conduction, and quality of life in myotonic dystrophy type 1 (DM1).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2011
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2011
CompletedFirst Submitted
Initial submission to the registry
July 20, 2011
CompletedFirst Posted
Study publicly available on registry
August 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2017
CompletedResults Posted
Study results publicly available
March 29, 2018
CompletedJune 19, 2018
May 1, 2018
5.7 years
July 20, 2011
March 1, 2018
May 21, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance
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.
Baseline to 6 months
Secondary Outcomes (5)
Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months
6 months
Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia
Baseline to 6 months
Mean Change From Baseline in Manual Muscle Testing (MMT) Score
Baseline to 6 months
Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring
Baseline to 6 Months
Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life
Baseline to 6 months
Study Arms (2)
Mexiletine
ACTIVE COMPARATOR20 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.
Sugar pill
PLACEBO COMPARATOR20 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.
Interventions
150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
Eligibility Criteria
You may qualify if:
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Ability to walk 30 feet (assistance with cane and/or leg bracing permitted)
- Presence of grip myotonia
You may not qualify if:
- Congenital DM1
- Treatment with Mexiletine within past 8 weeks
- Second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia
- Receiving another antimyotonia drug
- Liver or kidney disease requiring ongoing treatment
- Has a seizure disorder
- Is pregnant or lactating
- Had severe depression within 3 months or a history of suicide ideation
- Has any one of the following medical conditions: uncontrolled diabetes mellitus, congestive heart failure, symptomatic cardiomyopathy, symptomatic coronary artery disease, cancer (other than skin cancer) less than five years previously, multiple sclerosis, or other serious medical illness.
- Drug or alcohol abuse within 3 months
- Coexistence of another neuromuscular disease
- Is unable to give informed consent
- Severe arthritis or other medical condition (besides DM1) that would significantly impact ambulation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Rochester Medical Center, Department of Neurology
Rochester, New York, 14642, United States
Related Publications (2)
Logigian EL, Martens WB, Moxley RT 4th, McDermott MP, Dilek N, Wiegner AW, Pearson AT, Barbieri CA, Annis CL, Thornton CA, Moxley RT 3rd. Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1. Neurology. 2010 May 4;74(18):1441-8. doi: 10.1212/WNL.0b013e3181dc1a3a.
PMID: 20439846BACKGROUNDMoxley RT 3rd, Logigian EL, Martens WB, Annis CL, Pandya S, Moxley RT 4th, Barbieri CA, Dilek N, Wiegner AW, Thornton CA. Computerized hand grip myometry reliably measures myotonia and muscle strength in myotonic dystrophy (DM1). Muscle Nerve. 2007 Sep;36(3):320-8. doi: 10.1002/mus.20822.
PMID: 17587223BACKGROUND
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Charles Thornton, MD
- Organization
- University of Rochester
Study Officials
- PRINCIPAL INVESTIGATOR
Richard T. Moxley, III, MD
University of Rochester
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Associate Professor of Neurology
Study Record Dates
First Submitted
July 20, 2011
First Posted
August 1, 2011
Study Start
June 1, 2011
Primary Completion
February 1, 2017
Study Completion
March 1, 2017
Last Updated
June 19, 2018
Results First Posted
March 29, 2018
Record last verified: 2018-05