Trial · NCT01406873
Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
NCT01406873, Phase 2, Completed, University of Rochester; results first posted 2018-03-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1. Trials, record NCT01406873. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01406873
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
- NCT number
- NCT01406873
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 42 participants, actual
- Lead sponsor
- University of Rochester
- Conditions
- Myotonic Dystrophy
- Interventions
- Mexiletine, Placebo
- First posted
- 2011-08-01
- Last updated
- 2018-06-19
- Results first posted
- 2018-03-29
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance | 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. |
| Secondary | Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | 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. |
| Secondary | Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia | 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). |
| Secondary | Mean Change From Baseline in Manual Muscle Testing (MMT) Score | 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). Average MMT score is derived by averaging the individual MMT scores across the 26 individual muscles. |
| Secondary | Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring | 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. |
| Secondary | Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life | 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. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance | Baseline to 6 months | Intent to Treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication and have post-baseline efficacy assessment. Follow-up data was not collected on one participant in the mexiletine arm due to a broken foot. | Meters | MEAN |
| SECONDARY | Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | 6 months | Intent to Treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication and have post-baseline efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia | Baseline to 6 months | Intent to Treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication, and have post-baseline efficacy assessment at 6 months. Data was not analyzable on 3 people in the placebo arm. | Seconds | MEAN |
| SECONDARY | Mean Change From Baseline in Manual Muscle Testing (MMT) Score | Baseline to 6 months | Intent to Treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication and have post-baseline efficacy assessment. | Units on a scale | MEAN |
| SECONDARY | Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring | Baseline to 6 Months | Intent to Treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication and have post-baseline efficacy assessment. | Milliseconds | MEAN |
| SECONDARY | Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life | Baseline to 6 months | Intent to treat (ITT) population was defined as all participants who were randomized to the study, received at least one dose of study medication and have post-baseline efficacy assessment. | Units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance | Mexiletine | 17.44 | Standard Deviation 39.84 | Meters | |
| Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance | Placebo | 7.25 | Standard Deviation 38.93 | Meters | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Mexiletine | Study Drug Withdrawal | 2 | Participants | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Placebo | Study Drug Withdrawal | 0 | Participants | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Mexiletine | Study Drug Dose Reduction | 1 | Participants | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Placebo | Study Drug Dose Reduction | 0 | Participants | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Mexiletine | Study Drug Temporary Suspension | 1 | Participants | |
| Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months | Placebo | Study Drug Temporary Suspension | 0 | Participants | |
| Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia | Mexiletine | -1.01 | Standard Deviation 1.78 | Seconds | |
| Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia | Placebo | 0.43 | Standard Deviation 1.53 | Seconds |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Mexiletine | 21 | |
| Overall Study | STARTED | Placebo | 21 | |
| Overall Study | COMPLETED | Mexiletine | 20 | |
| Overall Study | COMPLETED | Placebo | 20 | |
| Overall Study | NOT COMPLETED | Mexiletine | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events160
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Cardiac disorders - Other, increased PVCs | Cardiac disorders | Mexiletine | 1 | 21 | 1 |
| other | Cardiac disorders - Other, increased PVCs | Cardiac disorders | Placebo | 0 | 21 | 0 |
| other | Chest pain - cardiac | Cardiac disorders | Mexiletine | 1 | 21 | 2 |
| other | Chest pain - cardiac | Cardiac disorders | Placebo | 1 | 21 | 2 |
| other | Ear pain | Ear and labyrinth disorders | Mexiletine | 0 | 21 | 0 |
| other | Ear pain | Ear and labyrinth disorders | Placebo | 1 | 21 | 1 |
| other | Tinnitus | Ear and labyrinth disorders | Mexiletine | 1 | 21 | 2 |
| other | Tinnitus | Ear and labyrinth disorders | Placebo | 0 | 21 | 0 |
| other | Blurred vision | Eye disorders | Mexiletine | 0 | 21 | 0 |
| other | Blurred vision | Eye disorders | Placebo | 1 | 21 | 1 |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Mexiletine | 0 | 21 |
| Total, all-cause mortality | Placebo | 0 | 21 |
| Total, other adverse events | Mexiletine | 21 | 21 |
| Total, other adverse events | Placebo | 20 | 21 |
| Total, serious adverse events | Mexiletine | 1 | 21 |
| Total, serious adverse events | Placebo | 1 | 21 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Mexiletine vs Placebo: deaths from any cause | Trial comparisons |
| Mexiletine vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01406873
GET https://conductscience.org/api/trials/v1/NCT01406873/results?page=2