Combined N-of-1 Trials Mexiletine vs Placebo in Patients With Non-Dystrophic Myotonia (NDM)

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.

Conditions

• Non Dystrophic Myotonia

Interventions

DRUG

Mexiletine

Mexiletine is a lidocaine-derivate and belongs to the class of 1B antiarrhythmic agents (Vaughan-Williams Classification of Antiarrhytmica). Class I antiarrhythmics have membrane-stabilizing properties. Drugs in this class work by interfering with the fast influx of sodium by inhibition of sodium ionchannels during the fast depolarization phase, thereby decreasing the maximal voltage and upshoot phase of the action potential. Mexiletine study-medication will be purchased from Stabilimento Chimico Farmaceutico Militare, Firenze, Italy.

DRUG

Placebo

Placebo tablets do not contain any active medicinal component.

Sponsors & Collaborators

• ZonMw: The Netherlands Organisation for Health Research and Development

  collaborator OTHER

• Radboud University Medical Center

  lead OTHER

Principal Investigators

• Prof. dr. BGM van Engelen, MD, PhD · Department of Neurology, Radboud University Nijmegen Medical Centre, the Netherlands

• Prof. dr. GJ van der Wilt, PhD · Department of Epidemiology, HTA and biostatistics, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. BC Stunnenberg, MD · Department of Neurology, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. W Woertman, PhD · Department of Epidemiologie, HTA and biostatistics, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. B Schouwenberg, MD · Department of Pharmacology and Toxicology, Radboud University Nijmegen Medical Centre, the Netherlands

• Dr. G Drost, MD, PhD · Department of Neurology, University Medical Centre Groningen, the Netherlands

• Drs. J Raaphorst, MD · Department of Neurology, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. N van Alfen, MD, PhD · Department of Neurology, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. J Timmermans, MD · Department of Cardiology, Radboud University Nijmegen Medical Centre, the Netherlands

• Drs. H Groenewoud, MSc · Department of Epidemiologie, HTA and biostatistics, Radboud University Nijmegen Medical Centre, the Netherlands

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

CROSSOVER

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2014-01-31

Primary Completion

2015-06-30

Completion

2015-06-30

Countries

• Netherlands

Study Locations