A Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders

Phase 1

• Conditions: Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2); ICD-10 code G71.1; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]; MedDRA version: 21.1Level: LLTClassification code 10028658Term: Myotonic disordersSystem Organ Class: 100000004852

• Registration Number: EUCTR2019-003758-97-FR
• Lead Sponsor: upin Europe GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-11-20
• Last Update Posted: 7 December 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. Patients previously enrolled in parent study PIP study 4 (MEX-NM-301) or PIP Study 5 (MEX-NM-302);
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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 listed in the Namuscla Summary of Product Characteristics (SmPC):
2.a. Hypersensitivity to the active substance, or to any of the excipients;
2.b. Hypersensitivity to any local anaesthetic;
2.c. Ventricular tachyarrhythmia;
2.d. 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);
2.e. QT interval > 450ms;
2.f. Myocardial infarction (acute or past), or abnormal Q-waves;
2.g. Symptomatic coronary artery disease;
2.h. Heart failure with ejection fraction <50%;
2.i. Atrial tachyarrhythmia, fibrillation or flutter;
2.j. Sinus node dysfunction (including sinus rate < 50 bpm);
2.k. Co-administration with medicinal products inducing torsades de pointes;
2.l. 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified