Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Lupin Europe GmbH

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

19 Nov 2024 → ongoing

Decision date (initial)

2024-11-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-513484-89-00

EudraCT number

2019-003758-97

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The aim of this study is to obtain additional information regarding the long-term safety and efficacy of mexiletine for the symptomatic treatment of myotonia in paediatric subjects who have completed the initial paediatric study MEX-NM-301.

Conditions and MedDRA coding

Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Patients previously completed the parent study PIP study 4 (MEX-NM-301) and tolerated the Mexiletine in the study.
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.
3. Patients continue to meet inclusion criteria of parent study (MEX-NM-301)

Exclusion criteria 8

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 described in the Namuscla Summary of Product Characteristics [SmPC])
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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
2. Incidence of adverse events of special interest (AESI)
3. Mean change in Visual Analogue Scale (VAS) or Faces score for muscle stiffness (myotonia severity) for children and adolescents aged 6 years to < 18 years (assessed every 3 months).
4. Score of handgrip myotonia as quantitatively measured using a commercially available grip dynamometer and computerized capture system in standardized conditions for children and adolescents aged 6 years to < 18 years (every 6 months).

Secondary endpoints 9

1. Mean change in VAS (8 to < 18 years) or Faces (6 to < 8 years) score for severity of muscle stiffness (if not a primary endpoint) pain, weakness and fatigue (every 3 months).
2. Clinical myotonia assessment (every 6 months) - Mean change in time to open the eyes after forced eye closure as measured on a stopwatch (when eyelid myotonia present)
3. Clinical myotonia assessment (every 6 months) - Clinical improvement in flexor myotonia (right hand flexor muscles)
4. Clinical myotonia assessment (every 6 months) - Mean change in time to perform Timed-up and go (TUG) test (patients aged 6 to <18 years only)
5. Mean change in health-related quality-of-life as measured by the Paediatric Quality of Life (PedsQL) score (secondary endpoint for patients aged 6 years to <18 years; every 6 months).
6. Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient, a parent or proxy and by the investigator. Measured every 6 months.
7. Mean change in Myotonia Behaviour Scale (MBS) scores (for patients aged 6 years to < 18 years; measured every 6 months).
8. Long-term safety of mexiletine - Changes in vital signs (every 3 months).
9. Long-term safety of mexiletine - Changes in clinical laboratory values (every 3 months).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Lupin Europe GmbH

Sponsor organisation

Lupin Europe GmbH

Address

Hanauer Landstrasse 139-143, Ostend Ostend

City

Frankfurt Am Main

Postcode

60314

Country

Germany

Scientific contact point

Organisation

Lupin Europe GmbH

Contact name

Alla Zozulya Weidenfeller

Public contact point

Organisation

Lupin Europe GmbH

Contact name

Alla Zozulya Weidenfeller

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

3 submissions — initial application plus substantial / non-substantial modifications