The Efficacy and Safety of Mexiletine PR compared to Mexiletine IR in NDM Patients (ACHILLES study)
Research type
Research Study
Full title
An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR compared to Mexiletine IR in Patients with Non-Dystrophic Myotonias (ACHILLES study)
IRAS ID
1010852
Contact name
Alla Zozulya Weidenfeller
Contact email
Sponsor organisation
Lupin Atlantis Holdings SA
Eudract number
2024-514970-27
Research summary
People over the age of 16 with non-dystrophic myotonias diagnosis confirmed genetically may join the study.
The study medication used in this trial is called Mexiletine prolonged released granules for oral suspension (mexiletine PR) or Mexiletine immediate release for oral administration (Mexiletine IR).
A prolonged release drug is a drug whose release is controlled over time, so that it diffuses more slowly and for longer in the body.
An immediate release drug is a drug formulated to release immediately after oral administration.
Sometimes because it is not known which way of treating patients is best, we need to make comparisons. People will be put into groups and then compared. The groups are selected by a computer which has no information about the individual – like a flip of a coin (called ‘randomization’). Subjects in each group then have a different treatment and these are compared.
This is a multicenter, open-label, randomized, cross-over study intended to evaluate the efficacy
and the safety of mexiletine PR (QD) vs mexiletine IR (TID) in patients with non-dystrophic
myotonias including myotonia congenita (MC), paramyotonia congenita (PC) and sodium
channel myotonia (SCM). The study will consist of a 4-week screening period followed by two
12-week treatment periods. Eligible patients will be randomized to receive mexiletine PR or
mexiletine IR for 12 weeks. After a wash out period of at least 7 days the patients will receive
the opposite treatment for 12 weeks.
A total of 24 patients are planned to be enrolled (with a target enrollment of 12 naïve to previous mexiletine treatment and 12 previously treated with mexiletine).
Safety assessments include patient- and physician-reported adverse event reporting, ECG, standard clinical laboratory evaluations, physical examinations, and vital signs. Efficacy assessments include patient-reported outcomes (PROs) and functional capacity outcome measures.REC name
London - Central Research Ethics Committee
REC reference
25/LO/0519
Date of REC Opinion
24 Jun 2025
REC opinion
Favourable Opinion