Myotonic DystrophyNCT04616807
An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders
An observational study of Myotonic Dystrophy, run by Lupin Ltd. at 6 sites in 3 countries.
Trial summary
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- Mexiletine
- How many people
- 53 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 17 Dec 2020to 16 Jan 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Study population: The study will target the enrolment of 50 patients who are treated with Namuscla.
Who may be able to join
- Adult, male or female patients with non-dystrophic myotonic disorders planned to be started on Namuscla according to the approved SmPC
- Patients already receiving Namuscla/mexiletine for the treatment of NDM; (for patients on mexiletine other than Namuscla, only those who switch to Namuscla will be enrolled).
- Patients who understand and are willing to provide informed consent.
Who cannot join
- Patients who are enrolled or participating in any other clinical trial for an investigational product. -
- Hypersensitivity to mexiletine, or to any of the excipients of Namuscla, or hypersensitivity to any local anaesthetic
- Ventricular tachyarrhythmia
- Atrial tachyarrhythmia, fibrillation or flutter
- Complete heart block (ie, third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 240 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
- Myocardial infarction (acute or past), or abnormal Q-waves
Show all 12Show fewer
- Symptomatic coronary artery disease
- Heart failure with reduced ejection fraction <50%
- Sinus node dysfunction (including sinus rate < 50 bpm)
- Patients receiving drugs that can induce torsades de pointes
- Patients receiving medicinal products with narrow therapeutic index (ie, theophylline, tizanidine, digoxin, lithium, phenytoin or warfarin)
- Patients who are pregnant or lactating.
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
France2 sites
CHRU Lille
Lille, 59000
Hôpital Universitaire de La Pitié Salpêtrière
Paris, Cedex, 13 75013
Germany2 sites
St. Josef-Hospital Klinikum der Ruhr Universitaet Bochum
Bochum, North Rhine-Westphalia, 44791
Universitätsklinikum Ulm, Klinik für Neurologie
Ulm, 89081
United Kingdom2 sites
Institute of Neurology
London, England, WC1N 3BG
Nottingham University Hospitals NHS Trust
Nottingham, England, NG7 2UH
About this study
Official title: An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.
Read the detailed description
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM. Namuscla should be prescribed as per the approved Summary of Product Characteristics (SmPC).
Adult patients with non-dystrophic myotonic disorders who have been prescribed Namuscla by the treating physician, and who meet the eligibility criteria will be enrolled in this study.
This includes:
- Patients newly initiated on Namuscla for the treatment of NDM (newly exposed)
- Patients already on Namuscla/ mexiletine at enrolment - For patients receiving mexiletine other than Namuscla, only those who switch to Namuscla will be included in the study.
Patients already being treated with Namuscla/ mexiletine at the time of enrolment will be considered for enrolment provided they meet the eligibility criteria.
The study will be initiated at specialized centres for the treatment of myotonic disorders ("reference centres") in the United Kingdom (UK), France, and Germany, depending on availability of Namuscla in the specific country. Depending on the enrolment and marketing status (availability) of Namuscla in other countries in the EU, inclusion of additional sites in other countries will be considered.
The study population will comprise patients who are diagnosed with non-dystrophic myotonic disorders and considered suitable candidates for the treatment by Namuscla by the investigators according to the approved SmPC. Patients will be enrolled over an approximate 2-year enrolment period and will be followed-up on-treatment for up to 3 years. Each enrolled patient will be observed for 3 years or until discontinuation (if discontinued early).
For all enrolled patients, the baseline would be the latest data available at the enrolment visit.
For the patients already on Namuscla, cumulative data (data related previous exposure as well as current data) will be collected for adverse events (AEs) on Namuscla treatment.
No drug will be supplied for this study; patients will receive medicines through local standard practices. All evaluations and investigations during the study will be performed according to the routine clinical practices and discretion of the treating physician.
- Conditions
- Myotonic Dystrophy
- Being tested
- Mexiletine
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MexiletineDrug
Also called Namuscla ™
Observational Study
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome 1 Proportion of patients with treatment-emergent AEs
Proportion of patients with treatment-emergent AEs ([TEAEs], including SAEs) from study enrolment to 6, 12, 24 and 36 months on Namuscla
Measured: Approximately 3 years
Primary Outcome 2 Proportion of patients requiring dose reduction or treatment discontinuation
Proportion of patients requiring dose reduction or treatment discontinuation due to AEs (including SAEs).
Measured: Approximately 3 years
Secondary outcomes (1)
Secondary Outcome Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI)
Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI) from study enrolment to 6, 12, 24, and 36 months
Measured: Approximately 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2020
- Study start17 Dec 2020
- Primary completion16 Jan 2026
- Study completion16 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lupin Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04616807
- Sponsor's protocol ID
- LUP/MEX/2018/001
- Status last verified
- Jun 2026
- First submitted
- 23 Oct 2020
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders
- ClinicalTrials.gov ID
- NCT04616807
- Acronym / sponsor ID
- LUP/MEX/2018/001
- Conditions
- Myotonic Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- Lupin Ltd.Industry (pharma, biotech, devices)
- Study start
- 17 Dec 2020 (actual)
- Primary completion
- 16 Jan 2026 (actual)
- Study completion
- 16 Jan 2026 (actual)
- First posted
- 5 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.
Detailed description
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM. Namuscla should be prescribed as per the approved Summary of Product Characteristics (SmPC).
Adult patients with non-dystrophic myotonic disorders who have been prescribed Namuscla by the treating physician, and who meet the eligibility criteria will be enrolled in this study.
This includes:
- Patients newly initiated on Namuscla for the treatment of NDM (newly exposed)
- Patients already on Namuscla/ mexiletine at enrolment - For patients receiving mexiletine other than Namuscla, only those who switch to Namuscla will be included in the study.
Patients already being treated with Namuscla/ mexiletine at the time of enrolment will be considered for enrolment provided they meet the eligibility criteria.
The study will be initiated at specialized centres for the treatment of myotonic disorders ("reference centres") in the United Kingdom (UK), France, and Germany, depending on availability of Namuscla in the specific country. Depending on the enrolment and marketing status (availability) of Namuscla in other countries in the EU, inclusion of additional sites in other countries will be considered.
The study population will comprise patients who are diagnosed with non-dystrophic myotonic disorders and considered suitable candidates for the treatment by Namuscla by the investigators according to the approved SmPC. Patients will be enrolled over an approximate 2-year enrolment period and will be followed-up on-treatment for up to 3 years. Each enrolled patient will be observed for 3 years or until discontinuation (if discontinued early).
For all enrolled patients, the baseline would be the latest data available at the enrolment visit.
For the patients already on Namuscla, cumulative data (data related previous exposure as well as current data) will be collected for adverse events (AEs) on Namuscla treatment.
No drug will be supplied for this study; patients will receive medicines through local standard practices. All evaluations and investigations during the study will be performed according to the routine clinical practices and discretion of the treating physician.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- The study will target the enrolment of 50 patients who are treated with Namuscla.
Key inclusion criteria (3 listed)
- Adult, male or female patients with non-dystrophic myotonic disorders planned to be started on Namuscla according to the approved SmPC
- Patients already receiving Namuscla/mexiletine for the treatment of NDM; (for patients on mexiletine other than Namuscla, only those who switch to Namuscla will be enrolled).
- Patients who understand and are willing to provide informed consent.
Key exclusion criteria (12 listed)
- Patients who are enrolled or participating in any other clinical trial for an investigational product. -
- Hypersensitivity to mexiletine, or to any of the excipients of Namuscla, or hypersensitivity to any local anaesthetic
- Ventricular tachyarrhythmia
- Atrial tachyarrhythmia, fibrillation or flutter
- Complete heart block (ie, third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 240 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MexiletineAlso: Namuscla ™Observational Study | Drug | – |
Primary outcome
Primary Outcome 1 Proportion of patients with treatment-emergent AEs
Time frame: Approximately 3 years
Proportion of patients with treatment-emergent AEs ([TEAEs], including SAEs) from study enrolment to 6, 12, 24 and 36 months on Namuscla
Primary Outcome 2 Proportion of patients requiring dose reduction or treatment discontinuation
Time frame: Approximately 3 years
Proportion of patients requiring dose reduction or treatment discontinuation due to AEs (including SAEs).
1 secondary outcome
Secondary Outcome Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI)
Time frame: Approximately 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myotonic Dystrophy, run by Lupin Ltd. at 6 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France, Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Outcome 1 Proportion of patients with treatment-emergent AEs (time frame: Approximately 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.