Non Dystrophic MyotoniaNCT07183059
A Single Center Prospective Study in an Estimated 570 Patients Who Underwent Genetic Screening at UZ Brussel in the Context of a Primary Cardiac Arrhythmia. Patients Showing a Variant Class 3,4 or 5 in SCN4A or CLCN1 Will Undergo a Clinical and Electrophysiological Review After IC.
A study testing Electromyography in people with Non Dystrophic Myotonia and Arrythmia, Cardiac, run by Universitair Ziekenhuis Brussel.
Trial summary
A prospective interventional single-center study will be conducted. The study includes clinically diagnosed Intramuros PCA-patients who underwent a PCA gene panel of 113 genes (see Appendix 1) in the UZ-Brussel since 2021. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Non Dystrophic Myotonia, Arrythmia, Cardiac
- What is being tested?
- Electromyography
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 570 participants
- Where
- No sites listed yet
- Study dates
- 15 Dec 2025to 30 Sep 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with PCA who underwent a genetic analysis with a PCA gene panel since 2021 (since the panel involves 112 genes including SCN4A).
- Male and female gender.
Who cannot join
- Genetic analysis before 2021
- Patients without cardiac screening in UZ Brussel
About this study
Official title: Exploring Overlap Between Primary Cardiac Arrhythmias and Non-dystrophic Myotonia: a Single Center Prospective Study.
A prospective interventional single-center study will be conducted. The study includes clinically diagnosed Intramuros PCA-patients who underwent a PCA gene panel of 113 genes (see Appendix 1) in the UZ-Brussel since 2021. In a retrospective part of the study, we will assess cardiac history, cardiac family history, cardiac exams and medical treatment and genetic data and family history. The prevalence of a class 3, 4 or 5 variant in the SCN4A and CLCN1 gene in the PCA-group will be compared to controls who underwent genetic screening for different causes, in which no association with muscular channelopathies is expected, without access to their medical file. In a prospective part of the study, patients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited for a one day visit for an interview, clinical neurological assessment and EMG. The aim of this second phase is to describe the clinical presentation of patients with concomitant PCA and non-dystrophic myotonia .
Read the detailed description
A prospective interventional single-center study will be conducted. The study includes clinically diagnosed Intramuros PCA-patients who underwent a PCA gene panel of 113 genes (see Appendix 1) in the UZ-Brussel since 2021.
Retrospective part:
The following retrospective data from the medical file will be analyzed:
- Informed consents signed before genetic analysis with PCA gene panel (Appendix 2).
- Assessment of cardiac history, cardiac family history, cardiac exams and medical treatment
- Assessment of genetic data and family history.
The PCA gene panel already contains the SCN4A gene since 2021. In case patients consented to scientific research or they consented to a wide genetic testing, additional genetic screening of the CLCN1 gene will be implemented. Those who did not consent previous to the PCA-gene panel analysis, will be contacted to sign a new informed consent if interested before additional screen-ing of the CLCN1 gene.
The prevalence of a class 3, 4 or 5 variant in the SCN4A and CLCN1 gene in the PCA-group will be compared to controls who underwent genetic screening for different causes, in which no associa-tion with muscular channelopathies is expected, without access to their medical file. These con-trols are the parents of patients with a congenital malformation syndrome or patients with a neu-rodevelopmental disorder. This to evaluate whether there's a higher prevalence of skeletal muscle channelopathies in patients with PCA compared to the general population.
Prospective part:
Patients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited by the department of Medical Genetics where they already have had a consultation, to the Neurology department for one visit on one day for the following as-sessments:
- Interview to assess the presence, onset and duration of myotonia or episodic weakness. Assessment of medical history, family history and medical treatment.
- Clinical neurological assessment for clinical myotonia.
- Electromyography (EMG) to detect the presence of myotonic discharges and using the Streib and Sun score for quantification(34) (see Appendix 3).
- Short exercise test in case of myotonic discharges on nEMG.
- In case of a diagnosis of a muscular channelopathy, assessment with.a QoL scale (see Ap-pendix 4).
The aim of this second phase is to describe the clinical presentation of patients with concomitant PCA and non-dystrophic myotonia . Since there is a known clinical variability between and within families with NDM, we aim to examine whether certain clinical features or severity of the disorder are linked to cardiac involvement.
Patients of the control group, who consented for scientific research and additional testing and who consented to be informed about incidental discoveries, who carry a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene, will be invited to the Neurology de-partment for a clinical evaluation outside of the study.
Patients with a variant class 3,4 or 5 in the SCN4A gene who did beforehand not consent for scien-tific research and/or for additional genetic testing are not additionally tested for variants in the CLCN1 gene but will be invited for a clinical evaluation after consent. If,, after clinical evaluation and EMG, they are diagnosed with myotonia, additional testing for a variant in the CLCN1 gene will be proposed.
- Conditions
- Non Dystrophic Myotonia, Arrythmia, Cardiac
- Being tested
- Electromyography
- Medical subject headings
- Arrhythmias, Cardiac
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElectromyographyDiagnostic test
we will perform questionnaire, clinical neurological exam, nEMG and if applicable short exercise test on patients with variants in genes associated with mus-cular channelopathies.
Safety Monitoring and reporting: Needle EMG is generally well-tolerated, but transient mild proce-dural pain and discomfort are widespread and the most frequent side effect that patients will ex-perience. Hematomas are infrequent and, in most cases, asymptomatic and selflimiting. Moreover, the bleeding risk is reduced by the investigator's experience, testing superficial muscles (avoiding complications like compartment syndrome), and by not conducting an EMG when patient is treated with anticoagulants. Infectious complications at the site of needle insertion are extremely infre-quent since disposable needle electrodes are used. Needle insertion is avoided in in-jured/potential infected skin and will not be performed in immune-compromised patients and endocarditis risk.
| Study group | Role | What they receive |
|---|---|---|
| Exploring overlap between primary cardiac arrhythmi-as and non-dystrophic myotonia: a single center | Experimental | Patients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited by the department of Medical Genetics where they already have had a consultation, to the Neurology department for one visit on one day for the following as-sessments:
* Interview to assess the presence, onset and duration of myotonia or episodic weakness. Assessment of medical history, family history and medical treatment.
* Clinical neurological assessment for clinical myotonia.
* Electromyography (EMG) to detect the presence of myotonic discharges and using the Streib and Sun score for quantification(34).
* Short exercise test in case of myotonic discharges on nEMG.
* In case of a diagnosis of a muscular channelopathy, assessment with.a QoL scale. Receives: Diagnostic Test: Electromyography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of variants of class 3,4 or 5 in the SCN4A gene and variants of class 3, 4 or 5 in het CLCN1 gene in patients presenting with PCA.
the prevalence of variants of class 3,4 or 5 in the SCN4A gene and variants of class 3, 4 or 5 in het CLCN1 gene in patients presenting with PCA compared to a control group
Measured: Day 1
Clinical presentation of NDMs in patients presenting with PCA and class 3,4 or 5 variants in SCN4A or CLCN1 gene.
the clinical presentation of NDMs in patients presenting with PCA and class 3,4 or 5 variants in SCN4A or CLCN1 gene.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2025
- Study start15 Dec 2025 (estimated)
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non Dystrophic MyotoniaStudies of the same condition, open and under way.
- More trials run by Universitair Ziekenhuis BrusselThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 570 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitair Ziekenhuis Brussel
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07183059
- Sponsor's protocol ID
- 25255_NDM-PCA studie
- Status last verified
- Aug 2025
- First submitted
- 4 Sep 2025
- Last updated
- 1 Dec 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploring Overlap Between Primary Cardiac Arrhythmias and Non-dystrophic Myotonia: a Single Center Prospective Study.
- ClinicalTrials.gov ID
- NCT07183059
- Acronym / sponsor ID
- 25255_NDM-PCA studie
- Conditions
- Non Dystrophic Myotonia, Arrythmia, Cardiac
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 570 participants (estimated)
- Lead sponsor
- Universitair Ziekenhuis BrusselUniversity, hospital or other
- Study start
- 15 Dec 2025 (estimated)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 19 Sep 2025 (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.
A prospective interventional single-center study will be conducted. The study includes clinically diagnosed Intramuros PCA-patients who underwent a PCA gene panel of 113 genes (see Appendix 1) in the UZ-Brussel since 2021. In a retrospective part of the study, we will assess cardiac history, cardiac family history, cardiac exams and medical treatment and genetic data and family history. The prevalence of a class 3, 4 or 5 variant in the SCN4A and CLCN1 gene in the PCA-group will be compared to controls who underwent genetic screening for different causes, in which no association with muscular channelopathies is expected, without access to their medical file. In a prospective part of the study, patients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited for a one day visit for an interview, clinical neurological assessment and EMG. The aim of this second phase is to describe the clinical presentation of patients with concomitant PCA and non-dystrophic myotonia .
Detailed description
A prospective interventional single-center study will be conducted. The study includes clinically diagnosed Intramuros PCA-patients who underwent a PCA gene panel of 113 genes (see Appendix 1) in the UZ-Brussel since 2021.
Retrospective part:
The following retrospective data from the medical file will be analyzed:
- Informed consents signed before genetic analysis with PCA gene panel (Appendix 2).
- Assessment of cardiac history, cardiac family history, cardiac exams and medical treatment
- Assessment of genetic data and family history.
The PCA gene panel already contains the SCN4A gene since 2021. In case patients consented to scientific research or they consented to a wide genetic testing, additional genetic screening of the CLCN1 gene will be implemented. Those who did not consent previous to the PCA-gene panel analysis, will be contacted to sign a new informed consent if interested before additional screen-ing of the CLCN1 gene.
The prevalence of a class 3, 4 or 5 variant in the SCN4A and CLCN1 gene in the PCA-group will be compared to controls who underwent genetic screening for different causes, in which no associa-tion with muscular channelopathies is expected, without access to their medical file. These con-trols are the parents of patients with a congenital malformation syndrome or patients with a neu-rodevelopmental disorder. This to evaluate whether there's a higher prevalence of skeletal muscle channelopathies in patients with PCA compared to the general population.
Prospective part:
Patients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited by the department of Medical Genetics where they already have had a consultation, to the Neurology department for one visit on one day for the following as-sessments:
- Interview to assess the presence, onset and duration of myotonia or episodic weakness. Assessment of medical history, family history and medical treatment.
- Clinical neurological assessment for clinical myotonia.
- Electromyography (EMG) to detect the presence of myotonic discharges and using the Streib and Sun score for quantification(34) (see Appendix 3).
- Short exercise test in case of myotonic discharges on nEMG.
- In case of a diagnosis of a muscular channelopathy, assessment with.a QoL scale (see Ap-pendix 4).
The aim of this second phase is to describe the clinical presentation of patients with concomitant PCA and non-dystrophic myotonia . Since there is a known clinical variability between and within families with NDM, we aim to examine whether certain clinical features or severity of the disorder are linked to cardiac involvement.
Patients of the control group, who consented for scientific research and additional testing and who consented to be informed about incidental discoveries, who carry a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene, will be invited to the Neurology de-partment for a clinical evaluation outside of the study.
Patients with a variant class 3,4 or 5 in the SCN4A gene who did beforehand not consent for scien-tific research and/or for additional genetic testing are not additionally tested for variants in the CLCN1 gene but will be invited for a clinical evaluation after consent. If,, after clinical evaluation and EMG, they are diagnosed with myotonia, additional testing for a variant in the CLCN1 gene will be proposed.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Exploring overlap between primary cardiac arrhythmi-as and non-dystrophic myotonia: a single center | Experimental | Diagnostic Test: ElectromyographyPatients with PCA carrying a variant class 3,4 or 5 in the SCN4A gene or a variant class 3, 4 or 5 in the CLCN1 gene will be invited by the department of Medical Genetics where they already have had a consultation, to the Neurology department for one visit on one day for the following as-sessments: * Interview to assess the presence, onset and duration of myotonia or episodic weakness. Assessment of medical history, family history and medical treatment. * Clinical neurological assessment for clinical myotonia. * Electromyography (EMG) to detect the presence of myotonic discharges and using the Streib and Sun score for quantification(34). * Short exercise test in case of myotonic discharges on nEMG. * In case of a diagnosis of a muscular channelopathy, assessment with.a QoL scale. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients with PCA who underwent a genetic analysis with a PCA gene panel since 2021 (since the panel involves 112 genes including SCN4A).
- Male and female gender.
Key exclusion criteria (2 listed)
- Genetic analysis before 2021
- Patients without cardiac screening in UZ Brussel
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 |
|---|---|---|
| Electromyographywe will perform questionnaire, clinical neurological exam, nEMG and if applicable short exercise test on patients with variants in genes associated with mus-cular channelopathies. Safety Monitoring and reporting: Needle EMG is generally well-tolerated, but transient mild proce-dural pain and discomfort are widespread and the most frequent side effect that patients will ex-perience. Hematomas are infrequent and, in most cases, asymptomatic and selflimiting. Moreover, the bleeding risk is reduced by the investigator's experience, testing superficial muscles (avoiding complications like compartment syndrome), and by not conducting an EMG when patient is treated with anticoagulants. Infectious complications at the site of needle insertion are extremely infre-quent since disposable needle electrodes are used. Needle insertion is avoided in in-jured/potential infected skin and will not be performed in immune-compromised patients and endocarditis risk. | Diagnostic test | Exploring overlap between primary cardiac arrhythmi-as and non-dystrophic myotonia: a single center |
Primary outcome
Prevalence of variants of class 3,4 or 5 in the SCN4A gene and variants of class 3, 4 or 5 in het CLCN1 gene in patients presenting with PCA.
Time frame: Day 1
the prevalence of variants of class 3,4 or 5 in the SCN4A gene and variants of class 3, 4 or 5 in het CLCN1 gene in patients presenting with PCA compared to a control group
Clinical presentation of NDMs in patients presenting with PCA and class 3,4 or 5 variants in SCN4A or CLCN1 gene.
Time frame: Day 1
the clinical presentation of NDMs in patients presenting with PCA and class 3,4 or 5 variants in SCN4A or CLCN1 gene.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2025.
- Individual participant dataSharing planned: Undecided
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Electromyography in people with Non Dystrophic Myotonia and Arrythmia, Cardiac, run by Universitair Ziekenhuis Brussel.
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 2 inclusion and 2 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: opening soon, as of the sponsor's last update on 1 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
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?
Prevalence of variants of class 3,4 or 5 in the SCN4A gene and variants of class 3, 4 or 5 in het CLCN1 gene in patients presenting with PCA. (time frame: Day 1). 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 1 Dec 2025; AzTrials last refreshed its copy on 6 Nov 2026.