An Open-label, non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children with Myotonic Disorders

upin Europe GmbH0 sites14 target enrollmentNovember 20, 2020

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

DrugsNamuscla

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2)
Sponsor: upin Europe GmbH
Enrollment: 14
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

November 20, 2020

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

upin Europe GmbH

Eligibility Criteria

Inclusion Criteria

•1\. Male or female patients aged \= 6 and \< 18 years who are able to comply with the study conditions;
•2\. A genetically confirmed diagnosis of NDM or DM (DM1or DM2\);
•3\. Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms);
•4\. No significant cardiac abnormalities as determined by a cardiologist’s assessment of the ECG and echocardiogram performed within 3 months prior to enrolment in the study. (if not done within 3 months before trial, ECG and echocardiogram assessments will be performed at screening);
•5\. No history of any significant liver disorder;
•6\. Patients receiving mexiletine treatment agree to stop treatment at least 7 days prior to initiation of treatment with Namuscla;
•7\. Patients receiving other antimyotonic treatment agree to stop treatment for at least 7 times the half\-life of respective drug;
•8\. Laboratory investigations for haematology, biochemistry, and urinalysis at screening are within normal range, or showing no clinically relevant abnormal values, as judged by the Investigator;
•9\. Female patients of childbearing potential must be using an acceptable form of birth control as determined by the Investigator (e.g., oral contraception, implantable, injectable/transdermal hormonal contraception, intrauterine device (IUD), barrier methods), tubal ligation or have a vasectomized partner or are practicing abstinence;
•10\. Patients able to provide assent to study participation and a parent or legal guardian able to sign written informed consent prior to study entry.

Exclusion Criteria

•1\. Any contra\-indication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC):
•1\.a. Hypersensitivity to the active substance, or to any of the excipients;
•1\.b. Hypersensitivity to any local anaesthetic;
•1\.c. Ventricular tachyarrhythmia;
•1\.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);
•1\.e. QT interval \> 450ms;
•1\.f. Myocardial infarction (acute or past), or abnormal Q\-waves;
•1\.g. Symptomatic coronary artery disease;
•1\.h. Heart failure with ejection fraction \<50%;
•1\.i. Atrial tachyarrhythmia, fibrillation or flutter;