NCT06523400

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

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)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 176 (estimated)
• Sponsor: Lupin Ltd. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

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)

Detailed description

This is a multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4- week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients in total complete/early terminate the study. In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo).

Conditions

• Myotonic Dystrophy

Interventions

Type	Name	Description
DRUG	Mexiletine granules for prolonged-release oral suspension	Mexiletine PR
DRUG	Placebo	Matching Placebo

Timeline

• Start date: 2025-02-13
• Primary completion: 2026-03-17
• Completion: 2026-04-22
• First posted: 2024-07-26
• Last updated: 2026-02-06

Locations

7 sites across 6 countries: Belgium, Denmark, Germany, Italy, Spain, United Kingdom