Active — Not RecruitingNot Applicable

An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders

France, Germany, United Kingdom53 participantsStarted 2020-12-17

Plain-language summary

This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Adult, male or female patients with non-dystrophic myotonic disorders planned to be started on Namuscla according to the approved SmPC
✓. Patients already receiving Namuscla/mexiletine for the treatment of NDM; (for patients on mexiletine other than Namuscla, only those who switch to Namuscla will be enrolled).
✓. Patients who understand and are willing to provide informed consent.

Exclusion criteria

✕. Patients who are enrolled or participating in any other clinical trial for an investigational product. -
✕. Hypersensitivity to mexiletine, or to any of the excipients of Namuscla, or hypersensitivity to any local anaesthetic
✕. Ventricular tachyarrhythmia
✕. Atrial tachyarrhythmia, fibrillation or flutter
✕. Complete heart block (ie, third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 240 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
✕. Myocardial infarction (acute or past), or abnormal Q-waves
✕. Symptomatic coronary artery disease
✕. Heart failure with reduced ejection fraction \<50%

What they're measuring

Primary Outcome 1 Proportion of patients with treatment-emergent AEs

Timeframe: Approximately 3 years

Primary Outcome 2 Proportion of patients requiring dose reduction or treatment discontinuation

Timeframe: Approximately 3 years

Trial details

NCT IDNCT04616807

SponsorLupin Ltd.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-12-19

View on ClinicalTrials.gov More Myotonic Dystrophy trials