Actively Recruiting
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
Led by Lupin Ltd. · Updated on 2026-02-06
176
Participants Needed
7
Research Sites
61 weeks
Total Duration
On this page
Sponsors
L
Lupin Ltd.
Lead Sponsor
L
Lupin Atlantis Holdings S.A.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of myotonic dystrophy type 1 (DM1) or type 2 (DM2) confirmed by genetic testing
- Ability and willingness to provide informed consent or assent (with parental consent if under 18)
- Understanding of study requirements and commitment to complete the 26-week treatment
- Male or non-pregnant female aged 16 years or older
- Body mass index between 18.5 and 30 kg/m2 and weight of at least 45 kg
- Female participants of childbearing potential must use effective birth control during the study and for at least 7 days after last dose
- No significant heart problems as assessed by a cardiologist
- Sufficient finger flexor strength to hold the dynamometer handle
- Presence of clinical handgrip myotonia with delayed grip relaxation of 3 seconds or more at screening
- Ability to walk independently for 10 meters (with cane, walker, or orthoses if needed)
- For DM1 patients only: muscular impairment rating scale score of 2, 3, or 4
You will not qualify if you...
- Pregnancy or breastfeeding
- Uncontrolled diabetes, cancer (except certain skin cancers within 5 years), multiple sclerosis, seizure disorders, or other serious illnesses
- Severe kidney impairment (glomerular filtration rate under 30 mL/min)
- Conditions affecting muscle function such as infections, trauma, fractures, or planned surgery
- Conditions impacting hand function like rheumatoid arthritis, Dupuytren's contracture, or hand deformities
- Severe arthritis or other conditions significantly affecting walking
- History of more than 5 falls in the past 12 months
- Elevated liver enzymes or other abnormal labs considered clinically significant
- Abnormal potassium or magnesium blood levels needing correction
- Use of mexiletine or anti-myotonic treatments within 4 weeks before starting the study
- Use of medications that increase cardiac risk
- Allergy to mexiletine or local anesthetics
- Participation in another interventional study within the last 3 months
- Being wheelchair-bound or bed-ridden
- Cardiac conditions including certain heart blocks, arrhythmias, heart failure, low heart function, or presence of defibrillators or pacemakers
- Use of medications that may cause dangerous heart rhythm effects
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
Laboratory for Muscle Diseases and Neuropathies
Leuven, Belgium
Actively Recruiting
2
Aarhus University Hospital
Aarhus, Denmark
Actively Recruiting
3
Ludug-Maximilians University
München, Germany
Actively Recruiting
4
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
Rome, Italy
Actively Recruiting
5
University Hospital of Madrid
Madrid, Spain
Actively Recruiting
6
Saint George's University Hospitals NHS Foundation Trust
London, United Kingdom
Actively Recruiting
7
University College Hospital
London, United Kingdom
Actively Recruiting
Research Team
N
Nikki Adetoro
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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