Combined N-of-1 Trials Mexiletine vs Placebo in Patients With Non-Dystrophic Myotonia (NDM) (NCT02045667) | Clinical Trial Compass
CompletedPhase 2
Combined N-of-1 Trials Mexiletine vs Placebo in Patients With Non-Dystrophic Myotonia (NDM)
Netherlands30 participantsStarted 2014-01
Plain-language summary
The main objective of this study is to explore whether multiple trials with individual patients (N-of-1 trials) can produce a reliable evidence base for coverage decisions on clinical and cost-effectiveness of drug treatment for patients with rare diseases. As a case study, we will study the clinical and cost-effectiveness of Mexiletine in patients with Non-Dystrophic myotonia. The results of this analysis will be compared with the results obtained from a recently published international, multi-centre, randomized, placebo-controlled trial of Mexiletine in patients with Non-Dystrophic Myotonia (clinicaltrials.gov Identifier: NCT00832000).
The secondary objective of this proposal is to assess whether mexiletine improves myotonia measured (both quantitatively and qualitative) in patients with non-dystrophic myotonia.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. At least 18 years of age
✓. Genetically confirmed diagnosis of NDMs
✓. Participation in the "Genetical variability of the Non-dystrophic Myotonia" study of J. Trip or a new patient with genetically confirmed NDM.
Exclusion criteria
✕. Inability or unwillingness to provide informed consent.
✕. Other neurological conditions that might affect the assessment of the study measurements.
✕. Genetic confirmed Myotonic Dystrophy type 1 (DM1) (CTG \> 50 repeats), or Myotonic Dystrophy type 2 (DM2).
✕. Patients with existing cardiac conduction defects, evidenced on ECG including but not limited to the following conditions: malignant arrhythmia or cardiac conduction disturbances (such as second degree AV block, third degree atrio-ventricular (AV) block, or prolonged QT interval \>500 ms or QRS duration \> 150 msec).
✕. Current use of the following antiarrhythmic medication for a cardiac disorder:flecainide acetate, encainide, disopyramide, procainamide, quinidine, propafenone or mexiletine.
What they're measuring
1
Change in patient-reported Stiffness on the IVR
Timeframe: Weeks 3-4 of each period - up to 44 weeks.
✕. Patients currently on medications for myotonia such as phenytoin and flecainide acetate within 5 days of enrollment, carbamazepine and mexiletine within 3 days of enrollment, or propafenone, procainamide, disopyramide, quinidine and encainide within 2 days of enrollment.
✕. Patients with renal or hepatic disease, heart failure, history of myocardial infarction, or seizure disorders.