Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01406873
• Date of registration: 20/07/2011
• Prospective Registration: No
• Primary sponsor: University of Rochester
• Public title: Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
• Scientific title: A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)
• Date of first enrolment: June 2011
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01406873
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard T. Moxley, III, MD
• Affiliation: University of Rochester

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myotonic Dystrophy

Intervention(s)

Drug: Mexiletine

Drug: Placebo

Primary Outcome(s)

Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance [Time Frame: Baseline to 6 months]

Secondary Outcome(s)

Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia [Time Frame: Baseline to 6 months]

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]

Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months [Time Frame: 6 months]

Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life [Time Frame: Baseline to 6 months]

Mean Change From Baseline in Manual Muscle Testing (MMT) Score [Time Frame: Baseline to 6 months]

Secondary ID(s)

3716

Funding Source: FDA/OOPD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 29/03/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01406873