Can a Muscle-Relaxing drug offer Long-Term relief for children with myotonia?

NCT ID NCT04622553

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 08, 2026 · Last updated Sep 09, 2026 · Updated 1 time

Summary

This trial evaluates the long-term safety and effectiveness of mexiletine in children aged 6 to 18 with myotonic disorders, conditions where muscles have trouble relaxing after contraction. Participants, who previously completed a study of mexiletine, receive the drug for about 8 weeks, with dose adjustments and regular heart monitoring. Researchers track side effects, muscle stiffness, and hand relaxation times to see if the drug remains safe and helpful over a longer period.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mexiletine (Namuscla), a drug that helps relax muscles after contraction
What this could lead to
If it works, this could support mexiletine as a long-term treatment to ease muscle stiffness in children with myotonic disorders.
What could go wrong
The trial is small and open-label, so results may not be conclusive. Mexiletine can affect heart rhythm, so heart monitoring is a key part of the study.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

12 people

The number who actually took part.

Started

Nov 2021

Finished

Jun 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patients previously completed the parent study PIP study 4 (MEX-NM-301) and tolerated the Mexiletine in the study. 2. Able and willing to provide assent to study participation and a parent or legal guardian willing to sign written informed consent prior to study entry. 3. Patients continue to meet inclusion criteria of parent study (MEX-NM-301): * No significant cardiac abnormalities as determined by a cardiologist's assessment of the ECG and Echocardiogram * No history or evidence of any significant liver disorder Laboratory investigations for haematology, biochemistry, and urinalysis at screening are within normal range, or showing no clinically relevant abnormal values, as judged by the Investigator * Female patients of childbearing potential must be using an acceptable form of birth control as determined by the Investigator (e.g., oral contraception, implantable, injectable/transdermal hormonal contraception, intrauterine device (IUD), barrier methods), tubal ligation or have a vasectomized partner or are practicing abstinence Exclusion Criteria: 1. Clinically significant laboratory abnormality, ECG or other clinical findings on physical examination indicative of a clinically significant exclusionary disease as determined by the investigator 2. Any contra-indication to mexiletine (as described in the Namuscla Summary of Product Characteristics \[SmPC\]) * Hypersensitivity to the active substance, or to any of the excipients * Hypersensitivity to any local anaesthetic * Ventricular tachyarrhythmia * Complete heart block (i.e., third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 200 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block), * QT interval \> 450ms * Myocardial infarction (acute or past), or abnormal Q-waves * Symptomatic coronary artery disease * Heart failure with ejection fraction \<50% * Atrial tachyarrhythmia, fibrillation or flutter * Sinus node dysfunction (including sinus rate \< 50 bpm) * Co-administration with medicinal products inducing torsades de pointes (class Ia, Ic, III antiarrhythmics): Co-administration of mexiletine and 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) increases the risk of potentially lethal torsades de pointes. * Co-administration with medicinal products with narrow therapeutic index 3. Co- administration with antiarrhythmics 4. Any other neurological or psychiatric condition that might affect the assessment of the study measurements 5. Any concurrent illness, or medications which could affect the muscle function 6. Seizure disorder, diabetes mellitus requiring treatment by insulin 7. Pregnant or breastfeeding 8. Concurrent participation in any other clinical trial.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Hôpital Necker-Enfants-Malades

    Paris, France

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