Study Comparing the Safety and Effectiveness of Mexiletine PR and Mexiletine IR for Adults with Non-Dystrophic Myotonias

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What is this study about?

This clinical trial is focused on studying a group of muscle disorders known as Non-Dystrophic Myotonias. These conditions cause muscle stiffness and difficulty relaxing muscles after use. The study will compare two forms of a medication called Mexiletine, which is used to treat these symptoms. The two forms being compared are Mexiletine PR (prolonged-release) and Mexiletine IR (immediate-release). The goal is to understand how safe and effective these medications are for treating muscle stiffness in adults with non-dystrophic myotonias, which include specific types like myotonia congenita, paramyotonia congenita, and sodium channel myotonia.

Participants in the study will receive either Mexiletine PR or Mexiletine IR over a period of 24 weeks. The study is designed as an open-label, randomized, cross-over trial, meaning that participants will switch between the two forms of the medication during the study. This approach helps researchers compare the effects of each medication form directly. The study will monitor the occurrence of any side effects and how well participants tolerate the medications. Additionally, the study will assess improvements in muscle stiffness and overall quality of life using various tests and questionnaires.

The primary focus is on the safety of the medications, looking at any adverse events that may occur during the treatment period. Secondary measures include video recordings of hand movements, quality of life assessments, and other tests to evaluate the impact of the medications on daily activities. The study aims to provide valuable information on the best treatment approach for managing symptoms in individuals with non-dystrophic myotonias.

1 introduction to the study

The study is designed to compare the safety and effectiveness of two forms of mexiletine for treating symptoms of non-dystrophic myotonias, which include conditions like myotonia congenita, paramyotonia congenita, and sodium channel myotonia.

Participants must have a confirmed diagnosis of one of these conditions and meet specific health criteria to be eligible.

2 medication administration

Participants will receive mexiletine in two forms: prolonged-release (PR) and immediate-release (IR).

The medications are administered orally, with dosages including 167 mg, 333 mg, and 500 mg for the prolonged-release form, and 167 mg for the immediate-release form.

The duration of treatment with each form is 12 weeks.

3 monitoring and assessments

Throughout the study, participants will be monitored for any adverse effects related to the medication.

Assessments will include video recordings of hand opening time, quality of life questionnaires, and various scales to measure myotonia symptoms and overall health.

4 completion of the study

The study is expected to conclude by April 30, 2026.

Upon completion, data will be analyzed to determine the safety and effectiveness of the two forms of mexiletine.

Who Can Join the Study?

  • Have a confirmed diagnosis of non-dystrophic myotonias, which includes conditions like myotonia congenita (MC), paramyotonia congenita (PC), and sodium channel myotonia (SCM).
  • Be a male or a non-pregnant female who is 16 years or older at the time of screening.
  • Have a Body Mass Index (BMI) between 18.5 and 30, and weigh at least 45 kilograms. BMI is a measure that uses your height and weight to work out if your weight is healthy.
  • If you are a female who can have children, you must use a highly effective form of birth control during the study and for at least 7 days after the last dose of the study drug.
  • Have no significant heart problems, as determined by a heart specialist’s assessment.
  • Have myotonic symptoms that are severe enough to need treatment.
  • Show signs of handgrip myotonia during screening, which means a delay of 3 seconds or more in relaxing your grip after squeezing your hand as hard as you can. This will be checked using a test called VHOT.

Who Cannot Join the Study?

  • Patients who are not adults cannot participate. This means only individuals who are 18 years or older can join.
  • Patients who do not have a diagnosis of Non-Dystrophic Myotonias (NDM) cannot participate. This includes specific types like myotonia congenita (MC), paramyotonia congenita (PC), and sodium channel myotonia (SCM).
  • Patients who are part of a vulnerable population may not be eligible. A vulnerable population includes groups that might need special protection, like those with certain disabilities or conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Universitaetsmedizin Goettingen Goettingen Germany
Katholieke Universiteit te Leuven Leuven Belgium
Université Libre de Bruxelles – Hôpital Erasme Brussels Belgium

Other Sites

Site Name City Country Status
Association Institut De Myologie Paris France
Hopital Beaujon Clichy France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
31.01.2025
France France
Recruiting
31.01.2025
Germany Germany
Not yet recruiting
31.01.2025
Italy Italy
Recruiting
31.01.2025

Trial locations

Mexiletine PR is a medication used in this study to help manage symptoms of myotonia, which is a condition that causes muscle stiffness. This version of mexiletine is designed to release the medication slowly over time, which may help maintain a more consistent level of the drug in the body and potentially reduce side effects.

Mexiletine IR is another form of the same medication used to treat myotonia. Unlike the PR version, this immediate-release form is designed to release the medication quickly into the body. This study aims to compare the safety and effectiveness of this form with the slow-release version in treating muscle stiffness in patients with non-dystrophic myotonias.

Investigated diseases:

Non-Dystrophic Myotonias – Non-dystrophic myotonias are a group of rare muscle disorders characterized by delayed relaxation of muscles after voluntary contraction. This condition leads to stiffness and difficulty in muscle movement, which can be triggered by sudden movements or changes in temperature. The symptoms can vary in severity and may affect different muscle groups, often causing discomfort and impacting daily activities. The condition is caused by genetic mutations affecting ion channels in muscle cells, leading to abnormal muscle excitability. It includes specific types such as myotonia congenita, paramyotonia congenita, and sodium channel myotonia, each with distinct genetic and clinical features. These disorders are typically present from childhood or adolescence and can persist throughout life.

Trial ID:
2024-514970-27-00
Protocol code:
MEX-NDM-301
Trial Phase:
Therapeutic confirmatory (Phase III)

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