Combined N-of-1 Trials Mexiletine vs Placebo in Patients With Non-Dystrophic Myotonia (NDM)
Status:
Completed
Trial end date:
2015-06-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Radboud University
Collaborator:
ZonMw: The Netherlands Organisation for Health Research and Development