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)
Who can participate
Age range16 Years
SexALL
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Inclusion criteria
✓. 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;
Exclusion criteria
✕. Are pregnant or lactating;
✕. Have any one of the following medical conditions: uncontrolled diabetes mellitus, cancer other than skin cancer less than five years previously (e.g., basal-cell carcinoma (BCC) and squamous-cell carcinoma (SCC) of skin allowed), multiple sclerosis, seizure disorders, or other serious medical illness;
To assess the efficacy of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2).
✕. Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;
✕. Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;
✕. Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
✕. High incidence of falls or fall-associated fractures (\>5 falls during the past 12 months);
✕. Preexisting elevated liver function tests \> 3 times the upper limit of normal (ULN) at screening (alanine transaminase (ALT)/aspartate transaminase (AST), gamma-glutamyl transferase (GGT)) and/or any abnormal chemistry, hematology or urine lab considered clinically significant by investigator;