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
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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)

Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:

• 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.-

Locations
Other Locations
Belgium
Laboratory for Muscle Diseases and Neuropathies
RECRUITING
Leuven
Denmark
Aarhus University Hospital
RECRUITING
Aarhus
Germany
Ludug-Maximilians University
RECRUITING
München
Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
RECRUITING
Rome
Spain
University Hospital of Madrid
RECRUITING
Madrid
United Kingdom
Saint George's University Hospitals NHS Foundation Trust
RECRUITING
London
University College Hospital
RECRUITING
London
Contact Information
Primary
Nikki Adetoro
nikkiadetoro@lupin.com
1 443-447-4534
Time Frame
Start Date: 2025-02-13
Estimated Completion Date: 2026-04-22
Participants
Target number of participants: 176
Treatments
Placebo_comparator: Placebo
0 mg (matching sachet volumes to low, medium and high dose active drug)
Active_comparator: Mexiletine prolonged-release (PR)
Mexiletine PR 167 mg (mexiletine HCl 200 mg) Mexiletine PR 333 mg (mexiletine HCl 400 mg) OR Mexiletine PR 500 mg (mexiletine HCl 600 mg)
Sponsors
Leads: Lupin Ltd.
Collaborators: Lupin Atlantis Holdings S.A.

This content was sourced from clinicaltrials.gov