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

NCT04616807 | Active Not Recruiting

• 1 other identifier: LUP/MEX/2018/001
• Study Type: observational
• Target: 53
• Locations: 3 countries
• Sites: 6

Brief 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.

Conditions

Myotonic Dystrophy

Outcome Measures

Primary Outcomes (2)

• Primary Outcome 1 Proportion of patients with treatment-emergent AEs

  Proportion of patients with treatment-emergent AEs (\[TEAEs\], including SAEs) from study enrolment to 6, 12, 24 and 36 months on Namuscla

  Approximately 3 years

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

  Proportion of patients requiring dose reduction or treatment discontinuation due to AEs (including SAEs).

  Approximately 3 years

Secondary Outcomes (1)

• Secondary Outcome Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI)

  Approximately 3 years

Interventions

Mexiletine DRUG

Observational Study

Also known as: Namuscla ™

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The study will target the enrolment of 50 patients who are treated with Namuscla.

You may qualify if:

• 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.

You may not qualify if:

• 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%
• Sinus node dysfunction (including sinus rate \< 50 bpm)
• Patients receiving drugs that can induce torsades de pointes
• Patients receiving medicinal products with narrow therapeutic index (ie, theophylline, tizanidine, digoxin, lithium, phenytoin or warfarin)
• Patients who are pregnant or lactating.

Sponsors & Collaborators

• Lupin Ltd.: lead

Study Sites (6)

Hôpital Universitaire de La Pitié Salpêtrière

Paris, Cedex, 13 75013, France

Location

CHRU Lille

Lille, 59000, France

Location

St. Josef-Hospital Klinikum der Ruhr Universitaet Bochum

Bochum, North Rhine-Westphalia, 44791, Germany

Location

Universitätsklinikum Ulm, Klinik für Neurologie

Ulm, 89081, Germany

Location

Institute of Neurology

London, England, WC1N 3BG, United Kingdom

Location

Nottingham University Hospitals NHS Trust

Nottingham, England, NG7 2UH, United Kingdom

Location

MeSH Terms

Conditions

Myotonic Dystrophy

Interventions

Mexiletine

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Propylamines; Amines; Organic Chemicals; Phenyl Ethers; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2020
• First Posted: November 5, 2020
• Study Start: December 17, 2020
• Primary Completion: December 19, 2025
• Study Completion: January 24, 2026
• Last Updated: July 25, 2025

Record last verified: 2025-07

Locations

FR (2); DE (2); GB (2)