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Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

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ClinicalTrials.gov Identifier: NCT01406873
Recruitment Status : Completed
First Posted : August 1, 2011
Results First Posted : March 29, 2018
Last Update Posted : March 29, 2018
Sponsor:
Information provided by (Responsible Party):
Chad Heatwole, University of Rochester

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Myotonic Dystrophy
Interventions: Drug: Mexiletine
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Mexiletine This group received 150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
Placebo This group received 150 mg/kg placebo capsules taken by mouth, three times daily for 6 months

Participant Flow:   Overall Study
    Mexiletine   Placebo
STARTED   21   21 
COMPLETED   20   20 
NOT COMPLETED   1   1 
Withdrawal by Subject                1                1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Mexiletine This group received 150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
Placebo This group received 150 mg/kg placebo capsules taken by mouth, three times daily for 6 months
Total Total of all reporting groups

Baseline Measures
   Mexiletine   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 21   21   42 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      0   0.0%      0   0.0%      0   0.0% 
Between 18 and 65 years      21 100.0%      21 100.0%      42 100.0% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 42.05  (11.54)   38.14  (9.78)   40.10  (10.75) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      17  81.0%      12  57.1%      29  69.0% 
Male      4  19.0%      9  42.9%      13  31.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      1   4.8%      1   4.8%      2   4.8% 
Not Hispanic or Latino      20  95.2%      20  95.2%      40  95.2% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      20  95.2%      20  95.2%      40  95.2% 
More than one race      1   4.8%      1   4.8%      2   4.8% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
     
United States   21   21   42 


  Outcome Measures

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

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

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

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

5.  Secondary:   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 ]

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


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Charles Thornton, MD
Organization: University of Rochester
phone: 585-275-2542
e-mail: Charles_Thornton@urmc.rochester.edu


Publications:

Responsible Party: Chad Heatwole, University of Rochester
ClinicalTrials.gov Identifier: NCT01406873     History of Changes
Other Study ID Numbers: 3716
Funding Source: FDA/OOPD ( Other Grant/Funding Number: R01FD003716 )
First Submitted: July 20, 2011
First Posted: August 1, 2011
Results First Submitted: March 1, 2018
Results First Posted: March 29, 2018
Last Update Posted: March 29, 2018