Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Richard Barohn, MD, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier:
NCT00832000
First received: January 27, 2009
Last updated: August 19, 2013
Last verified: August 2013
Results First Received: October 2, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Myotonia
Non-Dystrophic Myotonia
Interventions: Drug: Mexiletine
Drug: Placebo

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Eligible participants were at least 16 years of age, had clinical symptoms or signs of NDM, and myotonic potentials on electromyography. Participants were either enrolled in the CINCH NDM Natural History Study, or a new patient with genetically confirmed NDM, or with clinical features of NDM but negative myotonic dystrophy DNA testing.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients taking anti-myotonic agents were required to discontinue medications for a wash-out period equal to 7 times the half-life of elimination prior to their baseline visit. Participants were ineligible if they has specific contraindications to taking mexiletine (cardiac conduction defects, hepatic or renal disease, or heart failure).

Reporting Groups
  Description
Mexiletine Then Placebo

29 Participants will receive mexiletine for 4 weeks, then no intervention for 1 week, and finally placebo for 4 weeks.

Included in anaysis*: 28 patients

*Modified intention to treat analysis. 1 subject in each group not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period

Placebo Then Mexiletine

30 Participants will receive placebo for 4 weeks, then no intervention for 1 week, and finally mexiletine for 4 weeks.

Included in analysis* 29 patients

*Modified intention to treat analysis. 1 subject in each not included in primary analysis due to failure to make any calls to the IVR system for stiffness in either period.


Participant Flow:   Overall Study
    Mexiletine Then Placebo     Placebo Then Mexiletine  
STARTED     29 [1]   30 [2]
Crossed Over to Opposite Intervention     25 [2]   29 [1]
COMPLETED     23     29  
NOT COMPLETED     6     1  
Adverse Event                 2                 0  
Withdrawal by Subject                 1                 0  
No calls to IVR in either period                 1                 1  
No calls to IVR in period 2                 2                 0  
[1] Mexiletine 200 mg orally three times daily
[2] Placebo



  Baseline Characteristics
  Hide 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.
No text entered.

Reporting Groups
  Description
All Study Participants All participants received all inerventions; therefore, we combined all participants into one Arm/Group.

Baseline Measures
    All Study Participants  
Number of Participants  
[units: participants]
  59  
Age  
[units: participants]
 
<=18 years     1  
Between 18 and 65 years     56  
>=65 years     2  
Age  
[units: years]
Mean ± Standard Deviation
  42.9  ± 25  
Gender  
[units: participants]
 
Female     26  
Male     33  
Region of Enrollment  
[units: participants]
 
United States     31  
Canada     4  
United Kingdom     12  
Italy     12  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Patient-reported Stiffness on the IVR   [ Time Frame: Weeks 3-4 of each period ]

2.  Secondary:   Patient Reported Pain on the IVR   [ Time Frame: Weeeks 3-4 of each period ]

3.  Secondary:   Patient Reported Weakness on the IVR   [ Time Frame: Weeks 3-4 of each period ]

4.  Secondary:   Patient Reported Tiredness on the IVR   [ Time Frame: Weeks 3-4 of each period ]

5.  Secondary:   Quantitative Measure of Hand Grip Myotonia (Seconds)   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

6.  Secondary:   Compound Motor Action Potentials After Short Exercise Test   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

7.  Secondary:   Graded Myotonia by Needle Electromyography - Right Abductor Digiti Minimi   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

8.  Secondary:   Clinical Hand Grip Myotonia Evaluation (Seconds)   [ Time Frame: The end of period 1 (week 4) and the end of period 2 (week 9) ]

9.  Secondary:   Clinical Eye Closure Myotonia Evaluation (Seconds)   [ Time Frame: The end of period 1 (week 4) and the end of period 2 (week 9) ]

10.  Secondary:   Graded Myotonia by Needle Electromyography - Right Tibialis Anterior   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

11.  Secondary:   Compound Motor Action Potentials After Long Exercise Test   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

12.  Secondary:   Individualized Neuromuscular Quality of Life Scale - Summary Score   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]

13.  Secondary:   Short Form 36 - Physical Composite Score   [ Time Frame: Particiapnts who experienced weakness on mexiletine in either period 1 or period 2. ]

14.  Secondary:   Short Form 36 - Mental Composite Score   [ Time Frame: The end of period 1 (week 4) and period 2 (week 9) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Richard J. Barohn, MD
Organization: University of Kansas Medical Center
phone: 913-588-6095
e-mail: rbarohn@kumc.edu


No publications provided by University of Kansas

Publications automatically indexed to this study:

Responsible Party: Richard Barohn, MD, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier: NCT00832000     History of Changes
Obsolete Identifiers: NCT00721942
Other Study ID Numbers: 11050, FDA OPD RO1FD003454
Study First Received: January 27, 2009
Results First Received: October 2, 2012
Last Updated: August 19, 2013
Health Authority: United States: Food and Drug Administration