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Looking for participantsPhase3

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

Sponsor: Lupin Ltd.

NCT ID: NCT06523400

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Mexiletine granules for prolonged-release oral suspension (drug), Placebo (drug)
How long the study runs
Study runs about 28 months (dates as stated)
About the drug or intervention
Mexiletine granules for prolonged-release oral suspension — drug: Mexiletine PR · Placebo — drug: Matching Placebo
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
16 Years and over
Who
All
Number of participants
176
Started
2025-02-13
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for myotonic dystrophy
  • • Phase3 - 176 participants
  • • 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) Study website: https://myotonicdystrophystudy

Who can take part?

  • • Ages 16 Years and over
  • • Diagnosed with myotonic dystrophy

Where?

  • • London - Saint George's University Hospitals NHS Foundation Trust
  • • London - University College Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

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) Study website: https://myotonicdystrophystudy.com/

More detail

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). Study website: https://myotonicdystrophystudy.com/

Myotonic Dystrophy

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 16 Years and over
  • Who can join: All genders

Biomarkers mentioned

PR

What the study is looking for

  • ✓DM1 or DM2 diagnosis confirmed genetically;
  • ✓Ability to comprehend and willingness to sign an agreement to take part (ICF) or ICF of the parent(s)/legal guardian and...
  • ✓Ability to understand the study requirements including intention to stay in the study until the end-of-study visit...
  • ✓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;

Who cannot take part

  • ✗Are pregnant or lactating;
  • ✗Severe kidney impairment (glomerular filtration rate (GFR) \< 30 mL/min);
  • ✗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...
  • ✗Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
See the full criteria
Inclusion Criteria: 1. DM1 or DM2 diagnosis confirmed genetically; 2. 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); 3. Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment; 4. Male or non-pregnant female ≥16 years of age; 5. Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg; 6. 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; 7. No significant cardiac abnormalities as determined by a cardiologist's assessment; 8. Have sufficient finger flexor strength to grasp the handle of the dynamometer used to measure myotonia; 9. Presence of clinical handgrip myotonia (delayed relaxation of grip of ≥ 3 seconds after maximum voluntary contraction) at screening using VHOT; 10. Be able to walk independently 10 meters (cane, walker, orthoses allowed); 11. DM1 patients only - Muscular impairment rating scale (MIRS) score of 2, 3 or 4.- Exclusion Criteria: 1. Are pregnant or lactating; 2. 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; 3. Severe renal impairment (glomerular filtration rate (GFR) \< 30 mL/min); 4. Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery; 5. Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.; 6. Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation; 7. High incidence of falls or fall-associated fractures (\>5 falls during the past 12 months); 8. 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; 9. Serum potassium values \< 3.5 mmol/L or \> 5.0 mmol/L or serum magnesium values \< 1.7 mg/dL. Electrolytic imbalance such as hypokalaemia, hyperkalaemia or hypomagnesaemia may increase the proarrhythmic effects of mexiletine. Electrolyte imbalances need to be corrected before administering mexiletine and will be monitored throughout treatment. 10. Treatment with mexiletine within 4 weeks prior to baseline (Day 1); 11. Intake of any anti-myotonic treatment within 4 weeks prior to baseline (Day 1) or 5 half-lives, whichever is longer such as metformin, such as propafenone, flecainide, lamotrigine, carbamazepine or any other channel-blocker/ anticonvulsive drugs; 12. Use of any concomitant medications that could increase the cardiac risk; 13. Known allergy to mexiletine or any local anesthetics; 14. Participation in another interventional clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer; 15. Wheelchair-bound or bed-ridden; 16. Any cardiac safety associated condition including any of the following criteria detected by screening cardiac evaluations including 24-hr Holter monitor, echocardiogram and clinical evaluations: * PR interval ≥240 ms or QRS duration ≥120 ms on resting ECG * Personal history of 3rd degree or 2nd degree type 2 atrioventricular block or sinus node dysfunction with pauses ≥3 seconds, complete bundle branch block, bifascicular and trifascicular block or any heart block susceptible to evolve to complete heart block * Personal history of sustained atrial fibrillation, flutter or tachycardia (duration \>30 seconds) * Personal history of non-sustained (ventricular triplets or more) or sustained ventricular tachycardia * Myocardial infarction (acute or past) or coronary artery stenosis \>50%, presence of abnormal Q waves * New York Heart Association (NYHA) Class II to IV heart failure * Left ventricular systolic dysfunction with ejection fraction \<50% * Sinus node dysfunction (including ECG sinus rate \<50 beats per minute (BPM)) * Co-administration of antiarrhythmics inducing torsades de pointes (class Ia: quinidine, procainamide, disopyramide, ajmaline; class Ic: encainide, flecainide, propafenone, moricizine; class III: amiodarone, sotalol, ibutilide, dofetilide, dronedarone, vernakalant) * Co-administration of other classes of antiarrhythmics (class Ib: lidocaine, phenytoin, tocainide; class II: propranolol, esmolol, timolol, metoprolol, atenolol, carvedilol, bisoprolol, nebivolol; class IV: verapamil, diltiazem) * Patients with implantable cardioverter defibrillators (ICDs) and pacemakers are excluded * Presence of symptomatic coronary artery disease

Where Is This Study? (2 UK sites)

Saint George's University Hospitals NHS Foundation Trust

London, United Kingdom

Recruiting
Site contact (verified)

University College Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Rajinder Sidhu - Associate Director, Research Governance and Operations

uclh.jro-communications@nhs.net020 3447 9825

How to Get in Touch

Nikki Adetoro

Sponsor contact

CONTACT

1 443-447-4534 nikkiadetoro@lupin.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09