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 (Phase 3)
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)
• DM1 or DM2 diagnosis confirmed genetically;
• Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)/legal guardian and written assent from the patient (if patient \< 18 years of age);
• Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment;
• Male or non-pregnant female ≥16 years of age;
• Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg;
• Female patients of childbearing potential must be using a highly effective form of birth control for the duration of the study and for at least 7 days after last dose of study drug;
• No significant cardiac abnormalities as determined by a cardiologist's assessment;
• Have sufficient finger flexor strength to grasp the handle of the dynamometer used to measure myotonia;
• Presence of clinical handgrip myotonia (delayed relaxation of grip of ≥ 3 seconds after maximum voluntary contraction) at screening using VHOT;
⁃ Be able to walk independently 10 meters (cane, walker, orthoses allowed);
⁃ DM1 patients only - Muscular impairment rating scale (MIRS) score of 2, 3 or 4.-