Non-dystrophic MyotoniasPhase 3MYOMEXNCT02336477
Mexiletine and Non Dystrophic Myotonias
A Phase 3 study testing Mexiletine in people with Non-dystrophic Myotonias and Paramyotonia Congenita, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Non-dystrophic Myotonias, Paramyotonia Congenita, Myotonia Congenita
- What is being tested?
- Mexiletine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2011to Jan 2014
- Phase
- Phase 3
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetically definite MC and PC.
- Male and female participants, age between 18 and 65 who are able to comply with the study conditions.
- Participants who experience myotonic symptoms severe enough to justify treatment.
- The severity will be evaluated on:
- Clinical criteria: myotonia is considered as severe if it involves at least two segments (upper limb, lower limb or face)
- Disabling criteria: myotonia is considered severe if patients notice impacts on at least 3 of the 7 daily activities listed in the disabling section of the clinical myotonia scale (Annex 2).
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- Thus, patients who experience myotonic symptoms severe enough to justify treatment are those with myotonia that involves at least two segments and that have an impact on at least 3 daily activities.
- Participants who are drug naive or those who receiving mexiletine at Effective dosage and agreeing to stop treatment at least four days before inclusion .
- Pregnancy: Women: non-childbearing potential (i.e., postmenopausal or Surgically sterile) or must use a medically accepted contraceptive regimen; a pregnancy test will ensure that they are not pregnant.
- Normal cardiac exam performed by a cardiologist including EKG, and Cardiac ultrasound (if not done within 3 months before trial).
Who cannot join
- Intercurrent event which could interfere with the muscle function (infection,trauma, fracture, …)
- Coincidental renal, hepatic, respiratory, thyroid, other neuromuscular disease or heart disease that will contraindicate mexiletine or interfere with clinical evaluation.
- Use of any of the following medications that can interfere with muscle function :diuretics, anti epileptics (sodium channel blockers), antiarrhythmics, corticosteroids, beta-blockers,
- Allergy to mexiletine
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety of Mexiletine in Non-dystrophic Myotonias
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
Read the detailed description
- Specific aims
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover with wash-out trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
- Research design Because of their differing phenotypes, 12 Paramyotonia Congenita and 12 Myotonia Congenita subjects will be enrolled in a stratified trial
- Outcome variables
- primary outcome variable: the score of stiffness severity on a self-assessment scale (100 mm VAS) measured at baseline, at the end of phase I and phase II.
- secondary outcome measures:
- of efficacy:
- standardized EMG measures after repetitive short exercise test at cold and long exercise test
- chair test: time needed to stand up from a chair, walk around it and sit down again
- severity and disability scale of myotonia to be validated)
- quality of life scale (INQOL)
- rate of drop-outs
- of safety:
- adverse event frequency and severity
- EKG
- Perspectives
It is anticipated that the trial will:
- provide data that justify recommendations for treatment strategies for myotonic patients
- provide data to justify AFSAPPS regulatory approval of mexiletine for treatment of myotonia in order to guarantee the availability of the drug for patients
- develop standardized diagnostic and treatment assessment for non-dystrophic myotonias
- Being tested
- Mexiletine, placebo
- Keywords
- non-dystrophic myotonias, paramyotonia congenita, myotonia congenita, Mexiletine, Randomized clinical trial
- Medical subject headings
- Myotonic Disorders, Myotonia Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MexiletineDrug
- Blisters of 10 capsules of 200 mg mexiletine hydrochloride.
- Patients will receive gradual dose of the treatment as it would be done in clinical practice.
- Mexiletine will be started at 200 mg / day (1 capsule to be taken at the beginning of the meal) and will be increased by 200mg every 3 days to reach a maximum of 600mg / day in 3 taken in 1 week.
- The duration of each treatment period is 18 days minimum (maximum 22 days).
placeboDrug
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Mexiletine / Placebo Receives: Drug: Mexiletine; Drug: placebo |
| 2 | Experimental | Placebo / Mexiletine Receives: Drug: Mexiletine; Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
score of stiffness severity on a self-assessment scale (100 mm VAS)
Measured: 18 days
Secondary outcomes (5)
standardized EMG measures after repetitive short exercise test at cold and long exercise test
Measured: 18 days
chair test: time needed to stand up from a chair, walk around it and sit down again
Measured: 18 days
severity and disability scale of myotonia to be validated
Measured: 18 days
quality of life scale (INQOL)
Measured: 18 days
CGI efficacy (Clinical Global Impression- Efficacy index)
Measured: 18 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2015 (estimated)
- Study startJun 2011
- Primary completionJan 2014
- Study completionJan 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-dystrophic MyotoniasStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bertrand Fontaine, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de ParisSavine Vicart, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02336477
- Sponsor's protocol ID
- P091101
- Acronym
- MYOMEX
- Status last verified
- Jan 2015
- First submitted
- 8 Jan 2015
- Last updated
- 13 Jan 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Mexiletine in Non-dystrophic Myotonias
- ClinicalTrials.gov ID
- NCT02336477
- Acronym / sponsor ID
- MYOMEX / P091101
- Keywords
- non-dystrophic myotonias, paramyotonia congenita, myotonia congenita, Mexiletine, Randomized clinical trial
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Jun 2011
- Primary completion
- Jan 2014 (actual)
- Study completion
- Jan 2014 (actual)
- First posted
- 13 Jan 2015 (estimated)
- 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.
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
Detailed description
- Specific aims
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover with wash-out trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
- Research design Because of their differing phenotypes, 12 Paramyotonia Congenita and 12 Myotonia Congenita subjects will be enrolled in a stratified trial
- Outcome variables
- primary outcome variable: the score of stiffness severity on a self-assessment scale (100 mm VAS) measured at baseline, at the end of phase I and phase II.
- secondary outcome measures:
- of efficacy:
- standardized EMG measures after repetitive short exercise test at cold and long exercise test
- chair test: time needed to stand up from a chair, walk around it and sit down again
- severity and disability scale of myotonia to be validated)
- quality of life scale (INQOL)
- rate of drop-outs
- of safety:
- adverse event frequency and severity
- EKG
- Perspectives
It is anticipated that the trial will:
- provide data that justify recommendations for treatment strategies for myotonic patients
- provide data to justify AFSAPPS regulatory approval of mexiletine for treatment of myotonia in order to guarantee the availability of the drug for patients
- develop standardized diagnostic and treatment assessment for non-dystrophic myotonias
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Mexiletine, Drug: placeboMexiletine / Placebo |
| 2 | Experimental | Drug: Mexiletine, Drug: placeboPlacebo / Mexiletine |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Genetically definite MC and PC.
- Male and female participants, age between 18 and 65 who are able to comply with the study conditions.
- Participants who experience myotonic symptoms severe enough to justify treatment.
- Clinical criteria: myotonia is considered as severe if it involves at least two segments (upper limb, lower limb or face)
- Disabling criteria: myotonia is considered severe if patients notice impacts on at least 3 of the 7 daily activities listed in the disabling section of the clinical myotonia scale (Annex 2).
Key exclusion criteria (4 listed)
- Intercurrent event which could interfere with the muscle function (infection,trauma, fracture, …)
- Coincidental renal, hepatic, respiratory, thyroid, other neuromuscular disease or heart disease that will contraindicate mexiletine or interfere with clinical evaluation.
- Use of any of the following medications that can interfere with muscle function :diuretics, anti epileptics (sodium channel blockers), antiarrhythmics, corticosteroids, beta-blockers,
- Allergy to mexiletine
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 |
|---|---|---|
| Mexiletine* Blisters of 10 capsules of 200 mg mexiletine hydrochloride. * Patients will receive gradual dose of the treatment as it would be done in clinical practice. * Mexiletine will be started at 200 mg / day (1 capsule to be taken at the beginning of the meal) and will be increased by 200mg every 3 days to reach a maximum of 600mg / day in 3 taken in 1 week. * The duration of each treatment period is 18 days minimum (maximum 22 days). | Drug | 1, 2 |
| placebo | Drug | 1, 2 |
Primary outcome
score of stiffness severity on a self-assessment scale (100 mm VAS)
Time frame: 18 days
5 secondary outcomes
standardized EMG measures after repetitive short exercise test at cold and long exercise test
Time frame: 18 days
chair test: time needed to stand up from a chair, walk around it and sit down again
Time frame: 18 days
severity and disability scale of myotonia to be validated
Time frame: 18 days
quality of life scale (INQOL)
Time frame: 18 days
CGI efficacy (Clinical Global Impression- Efficacy index)
Time frame: 18 days
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 13 Jan 2015.
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Myotonia
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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.
Non Dystrophic Myotonia, Arrythmia, Cardiac
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.
Bertrand Fontaine, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
Savine Vicart, MD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mexiletine in people with Non-dystrophic Myotonias and Paramyotonia Congenita, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 10 inclusion and 4 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 13 Jan 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
score of stiffness severity on a self-assessment scale (100 mm VAS) (time frame: 18 days). 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 13 Jan 2015.