The Efficacy and Safety of Once Daily Mexiletine PR in Patients with DM1 and DM2 (HERCULES study)
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)
IRAS ID
1009815
Contact name
Chris Turner
Contact email
Sponsor organisation
Lupin Atlantis Holdings SA
Clinicaltrials.gov Identifier
Research summary
The study medication used in this trial is called mexiletine prolonged released granules for oral suspension (mexiletine PR) or matching placebo.
A placebo is defined as a treatment that has no pharmacological effect of its own. All other details would be the same as that of the investigational product. 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.
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. Neither the patient nor the study doctor chooses the assigned group. There is an equal chance of being in either group.
This is a double-blind trial, thus neither the patient nor the study doctor knows which treatment group is assigned. (if the doctor needs to find out they can do so).
Treatment will be given orally (by mouth). It is supplied as off-white to yellow coloured granular powder in unit dose sachet and taken only as instructed.
Currently, there is no drug for myotonia symptoms, therefore there is an unmet need for an approved drug in this DM population.
Mexiletine PR is an investigational (experimental) drug that has not been approved by Medicines and Healthcare products Regulatory Agency (MHRA) or any other regulatory authorities in the world.
However, NaMuscla (mexiletine) has been approved for use of symptomatic treatment of myotonia in the adult non-dystrophic myotonic disorders (NDM) patient population.
The goal of this well-controlled 6-month treatment study is to show that the benefits of using mexiletine outweigh any potential risks for patients with DM1 and DM2.REC name
London - Dulwich Research Ethics Committee
REC reference
24/LO/0453
Date of REC Opinion
1 Aug 2024
REC opinion
Further Information Favourable Opinion