The Long-Term Safety of Mexiletine PR in Patients with Myotonic Dystrophy DM1 and DM2 (ATLAS Study)
Research type
Research Study
Full title
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients with Myotonic Dystrophy Type 1 and Type 2 who have completed the MEX-DM-302 Study
IRAS ID
1009816
Contact name
Chris Turner
Contact email
Sponsor organisation
Lupin Atlantis Holdings SA
Research summary
Mexiletine PR is an investigational (experimental) that has not been approved by European Medicines Agency 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. NaMuscla was shown to be effective at easing the symptoms of myotonia in patients with NDM, thus improving their quality of life. This is an open-label study in which all participants will be offered a treatment with the investigational drug, mexiletine PR, not placebo for the duration of 18 months.
The goal of this well-controlled 18-month treatment study is to show that the benefits of using mexiletine outweigh any potential risks for patients with DM1 and DM2. Mexiletine PR powder for oral suspension has been developed to allow better swallowability, once daily dosing, and easy dose adjustment to support better clinical outcomes thanks to improved compliance to treatment intake.
All patients completed HERCULES study at approximately 12 experienced investigational sites in Europe and UK are planned to be rolled over into this open label long term follow up ATLAS study. Eligible patients will all be assigned mexiletine PR for 18 months.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
24/EE/0136
Date of REC Opinion
26 Jul 2024
REC opinion
Further Information Favourable Opinion