NCT04624750 is a Phase 3 clinical trial of Mexiletine in Myotonic Dystrophy, sponsored by Lupin Ltd..

Who is sponsoring NCT04624750?

NCT04624750 is sponsored by Lupin Ltd..

What drugs are being tested in NCT04624750?

Interventions in NCT04624750: Mexiletine.

What condition does NCT04624750 study?

NCT04624750 studies Myotonic Dystrophy.

Is NCT04624750 still recruiting?

NCT04624750 is currently completed. Last updated 25 November 2024.

Last reviewed 2024-11-25 · How we verify

NCT04624750

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Open Label Study in Adolescents and Children With Myotonic Disorders

Completed Phase 3 Last updated 25 November 2024

What this trial tests

Phase 3 trial testing Mexiletine in Myotonic Dystrophy in 12 participants. Completed in 8 August 2024.

Timeline

3 September 2021

Primary endpoint
14 June 2024

8 August 2024

Quick facts

Lead sponsor	Lupin Ltd.
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	3 September 2021
Primary completion	14 June 2024
Estimated completion	8 August 2024
Sites	1 location across France

Drugs / interventions tested

Mexiletine (MEXILETINE) — full drug profile →

Conditions studied

Myotonic Dystrophy — all drugs for Myotonic Dystrophy →

Sponsor

Lupin Ltd. — full company profile →

Who can join

Adults 6 to 18, any sex, with Myotonic Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to \<18 years of age) with myotonic disorders.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

Myotonic dystrophy type 1 drug development: A pipeline toward the market.
Pascual-Gilabert M, López-Castel A, Artero R. · · 2021 · cited 39× · PMID 33798646 · DOI 10.1016/j.drudis.2021.03.024
Recent Progress and Challenges in the Development of Antisense Therapies for Myotonic Dystrophy Type 1.
De Serres-Bérard T, Ait Benichou S, Jauvin D, Boutjdir M, et al · · 2022 · cited 17× · PMID 36362145 · DOI 10.3390/ijms232113359
Multisystem Symptoms in Myotonic Dystrophy Type 1: A Management and Therapeutic Perspective.
Kuntawala DH, Vitorino R, Cruz AC, Martins F, et al · · 2025 · cited 4× · PMID 40508159 · DOI 10.3390/ijms26115350
Drug treatment for myotonia.
Spillane J, Trip J, Drost G, Faber CG, et al · · 2025 · PMID 40197813 · DOI 10.1002/14651858.cd004762.pub3
Pharmacological therapy of non-dystrophic myotonias.
Saltarella I, Laghetti P, Dell'Atti S, Altamura C, et al · · 2025 · PMID 40183437 · DOI 10.36185/2532-1900-1026

Verify or expand the search:

Other trials of Mexiletine

Trials testing the same drug.

NCT07154654 — Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders · recruiting
NCT05619120 — The Effect of Late Na Current Blocker Mexiletine on Giant T-wave Electrical Alternans(STOP-TWA) · NA · recruiting
NCT04622553 — Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study. · NA · active not recruiting
NCT04616807 — An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders · active not recruiting

Other recruiting trials for Myotonic Dystrophy

Currently open trials in the same condition.

NCT07321977 — Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular D · NA · recruiting
NCT06844214 — A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Ge · Phase 1, PHASE2 · recruiting
NCT06523400 — The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 · Phase 3 · recruiting
NCT04616807 — An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders · active not recruiting

Other Lupin Ltd. trials

Trials by the same sponsor.

NCT07154654 — Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders · recruiting
NCT06549400 — An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With · Phase 3 · enrolling by invitation
NCT07097701 — An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexileti · Phase 3 · recruiting
NCT06523400 — The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 · Phase 3 · recruiting
NCT04622553 — Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study. · NA · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04624750 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Lupin Ltd.
Last refreshed: 25 November 2024

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04624750.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing