Phase 3 Muscular Dystrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have been diagnosed with Myotonic Dystrophy Type 1 (DM1) or Type 2 (DM2) confirmed by genetic testing.
• People who are able to understand and willing to sign a consent form (or, for those under 18, whose parent or legal guardian signs the form and who themselves provide written agreement).
• People who are willing and able to stay in the study for the full 26 weeks until the final visit.
• People who are male or non-pregnant female and are 16 years of age or older.
• People whose Body Mass Index (BMI) is between 18.5 and 30, and who weigh at least 45 kg.
• Female participants who could become pregnant must be using a highly effective form of contraception throughout the study and for at least 7 days after the last dose of the study drug.
• People who a heart specialist has confirmed have no significant heart abnormalities.
• People who have enough strength in their finger muscles to grip the handle of the testing device used to measure muscle stiffness.
• People who show a specific type of hand muscle stiffness at the screening visit — where the hand takes 3 seconds or longer to relax after gripping as hard as possible.
• People who are able to walk 10 metres on their own (walking aids such as a cane, walker, or leg braces are allowed).
• For DM1 patients only: people whose muscle impairment level falls within a specific range on a standard rating scale (score of 2, 3, or 4 on the Muscular Impairment Rating Scale) (confirm with trial site).

Who may not be able to join:

• People who are pregnant or breastfeeding.
• People who have any of the following conditions: uncontrolled diabetes, a cancer diagnosis within the past five years (skin cancers such as basal-cell or squamous-cell carcinoma of the skin may be allowed), multiple sclerosis, seizure disorders, or another serious illness.
• People with severely reduced kidney function (a filtering rate below 30 mL/min).
• People with conditions that could affect muscle function, such as active infections, recent injuries, broken bones, or planned surgery.
• People with conditions affecting hand function, such as rheumatoid arthritis, Dupuytren's contracture, or hand deformities.
• People with severe arthritis or another condition (not related to DM1/DM2) that significantly affects their ability to walk.
• People who have had more than 5 falls in the past 12 months, or fall-related broken bones.
• People whose liver function tests at screening are more than 3 times the upper normal limit, or who have other blood or urine test results considered clinically significant by the investigating doctor.
• People whose blood potassium or magnesium levels fall outside a specific normal range at screening (confirm with trial site for exact values).
• People who have taken mexiletine (the study drug) within 4 weeks before the start of the study.
• People who have taken any anti-myotonia medications within 4 weeks before the start of the study (or within 5 half-lives of the drug, whichever is longer) — this includes drugs such as metformin, propafenone, flecainide, lamotrigine, carbamazepine, or similar medicines.
• People who are currently taking any other medications that could increase heart risk.
• People who have a known allergy to mexiletine or any local anaesthetic.
• People who have taken part in another interventional clinical trial within the past 3 months, or within 5 half-lives of the investigational drug used in that trial, whichever is longer.
• People who are wheelchair-bound or bed-ridden.
• People who have certain heart-related conditions identified during screening tests (including a 24-hour heart monitor, echocardiogram, and clinical review), including but not limited to:
  • Specific abnormal readings on a resting ECG (heart tracing) relating to electrical conduction timing.
  • A personal history of certain types of heart block or problems with the heart's natural pacemaker function.
  • A personal history of sustained irregular heart rhythms lasting more than 30 seconds.
  • A personal history of certain types of abnormal rapid heart rhythms (ventricular tachycardia).
  • A history of heart attack or significant narrowing of the heart's arteries (more than 50%).
  • Heart failure classified as moderate to severe (Class II to IV on a standard scale).
  • Reduced pumping function of the heart (ejection fraction below 50%).
  • A slow resting heart rate of fewer than 50 beats per minute, or other problems with the heart's natural pacemaker.
  • Current use of certain heart rhythm medications that may interact with the study drug (confirm specific drug names with trial site).
  • Having an implanted heart device such as a defibrillator or pacemaker.
  • Symptoms of coronary artery disease.

Important: Always verify eligibility with the trial site directly before applying.