Mexiletine HCl is backed by numerous clinical trials ranging from Phase I to Phase IV studies that evaluate its efficacy across cardiac disorders and neuromuscular conditions, including myotonic dystrophy, amyotrophic lateral sclerosis, and non-dystrophic myotonia. Sponsored by both academic medical centers and industry partners, this research portfolio encompasses active, recruiting, and completed protocols examining key endpoints such as muscle cramp management and cortical hyperexcitability.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06549400 | ENROLLING_BY_INVITATION | Myotonic Dystrophy |
Lupin Ltd. |
2025-09-12 | PHASE3 |
| NCT06862596 | RECRUITING | Spinal and Bulbar Muscular Atrophy |
Masahisa Katsuno |
2025-02-28 | PHASE2; PHASE3 |
| NCT06669299 | RECRUITING | Ventricular Tachycardia (VT) |
IRCCS Ospedale San Raffaele |
2025-06-12 | |
| NCT07097701 | RECRUITING | Myotonia |
Lupin Ltd. |
2025-09-03 | PHASE3 |
| NCT06523400 | RECRUITING | Myotonic Dystrophy |
Lupin Ltd. |
2025-02-13 | PHASE3 |
| NCT04622553 | ACTIVE_NOT_RECRUITING | Myotonic Disorders |
Lupin Ltd. |
2021-11-05 | |
| NCT05619120 | UNKNOWN | Giant T-wave Electrical Alternans |
First Affiliated Hospital Xi'an Jiaotong University |
2023-01-01 | |
| NCT04700046 | WITHDRAWN | Myotonic Dystrophy Type 1 and Type 2 |
Lupin Ltd. |
2021-09-03 | PHASE3 |
| NCT04624750 | COMPLETED | Myotonic Dystrophy |
Lupin Ltd. |
2021-09-03 | PHASE3 |
| NCT05017155 | UNKNOWN | Non-Dystrophic Myotonia |
University College, London |
2021-08-12 | PHASE3 |
| NCT02781454 | COMPLETED | Sporadic Amyotrophic Lateral Sclerosis |
University of Washington |
2016-10 | PHASE2 |
| NCT02260388 | COMPLETED | Cryptogenic Sensory Polyneuropathy |
University of Kansas Medical Center |
2014-10 | PHASE4 |
| NCT01406873 | COMPLETED | Myotonic Dystrophy |
University of Rochester |
2011-06 | PHASE2 |
| NCT02561702 | COMPLETED | Charcot Marie Tooth Disease |
University of Rochester |
2015-09 | PHASE2 |
| NCT01811355 | COMPLETED | Muscle Cramps in Amyotrophic Lateral Sclerosis |
Bjorn Oskarsson, MD |
2013-05 | PHASE4 |
| NCT02045667 | COMPLETED | Non Dystrophic Myotonia |
Radboud University Medical Center |
2014-01 | PHASE2 |
| NCT01849770 | COMPLETED | Sporadic Amyotrophic Lateral Sclerosis |
University of Washington |
2013-07 | PHASE2 |
| NCT02308748 | COMPLETED | Drug-induced QT Prolongation; Pharmacokinetics; Pharmacodynamics |
Food and Drug Administration (FDA) |
2014-05 | PHASE1 |
| NCT02336477 | COMPLETED | Non-dystrophic Myotonias; Paramyotonia Congenita; Myotonia Congenita |
Assistance Publique - Hôpitaux de Paris |
2011-06 | PHASE3 |
| NCT01717404 | COMPLETED | IBS |
Mayo Clinic |
2012-10 | PHASE1 |
| NCT00832000 | COMPLETED | Myotonia; Non-Dystrophic Myotonia |
Richard Barohn, MD |
2008-12 | PHASE2 |
| NCT00000464 | COMPLETED | Arrhythmia; Cardiovascular Diseases; Heart Arrest; Heart Diseases; Myocardial Infarction; Ventricular Fibrillation |
University of Washington |
1987-04 | PHASE3 |
| NCT00000518 | COMPLETED | Arrhythmia; Cardiovascular Diseases; Death, Sudden, Cardiac; Heart Diseases; Tachycardia, Ventricular; Ventricular Arrhythmia; Ventricular Fibrillation |
University of Utah |
1985-07 | PHASE3 |
| NCT00004647 | COMPLETED | Pain; Diabetic Neuropathies; Paresthesia |
National Center for Research Resources (NCRR) |
1993-03 | PHASE3 |
| NCT00383682 | COMPLETED | Amputation; Chronic Pain |
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
1997-07 | PHASE2 |
| NCT00000793 | COMPLETED | HIV Infections; Peripheral Nervous System Disease |
National Institute of Allergy and Infectious Diseases (NIAID) |
PHASE2 | |
| NCT00001784 | COMPLETED | Blepharospasm; Dystonia; Spasm |
National Institute of Neurological Disorders and Stroke (NINDS) |
1998-07 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-01-26)
Check the
Mexiletine HCl
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
Note: Technical data last updated: Sep 1, 2026.