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Tests treatment safety and results for Myotonic Dystrophy
Official title The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
ClinicalTrials.gov ID: NCT06523400
What this study is testing
What is Mexiletine granules for prolonged-release oral suspension?
Mexiletine granules for prolonged-release oral suspension is an investigational medicine, given as an once-daily, being studied as a potential treatment for myotonic dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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)
- Phase 3: a large, late-stage study
- Time commitment: about 26 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 and older
You may be able to join if
- 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...
- 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;
You likely can't join if
- 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...
- 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;
- Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity...
- Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
See the full eligibility criteria
- 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.-
- 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...
- 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;
- Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;
- Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
- High incidence of falls or fall-associated fractures (\>5 falls during the past 12 months);
- 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...
- Serum potassium values \ 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...
- Treatment with mexiletine within 4 weeks prior to baseline (Day 1);
- 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...
- Use of any concomitant medications that could increase the cardiac risk;
- Known allergy to mexiletine or any local anesthetics;
- Participation in another treatment clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer;
- Wheelchair-bound or bed-ridden;
- 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...
- 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...
- 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...
- Patients with implantable cardioverter defibrillators (ICDs) and pacemakers are excluded
- Presence of symptomatic coronary artery disease
The study team makes the final eligibility decision.
Where it's taking place
- Leuven, Belgium
- Aarhus, Denmark
- München, Germany
- Rome, Italy
- Madrid, Spain
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 26 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Leuven, Belgium; Aarhus, Denmark; München, Germany; Rome, Italy; Madrid, Spain; London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.