The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2

ConditionMyotonic Dystrophy

Trial statusRecruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age16+

SponsorLupin Ltd.

About this trial

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)

Eligibility criteria

Qualifiers

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;

Disqualifiers

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;

Severe renal impairment (glomerular filtration rate (GFR) < 30 mL/min);

Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;