Non-Dystrophic MyotoniaNCT05639257
Treatment of Myotonia - Lamotrigine Versus Namuscla
A study testing Lamotrigine and Namuscla in people with Non-Dystrophic Myotonia, run by Grete Andersen, MD at 1 site in Denmark.
Trial summary
In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-Dystrophic Myotonia
- What is being tested?
- Lamotrigine and Namuscla
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteDenmark
- Study dates
- 5 Dec 2022to 1 Apr 2024
- Phase
- Interventional
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Capable adult women and men (age ≥ 18 years).
- Diagnosed with Myotonia Congenita (Becker and Thomsen type), Paramyotonia Congenita, or Hyperkalemic periodic paralysis.
- Myotonia under treatment or which significantly limits the daily activities (MBS> 2).
Who cannot join
- Allergy to lamotrigine, mexiletine, or the inactive ingredients in trial medication.
- Disease, which is affected by trial medication such as heart disease (ischemia and arrhythmia), epilepsy, and significant renal or hepatic failure.
- Treatment that, in the opinion of the project manager, can affect the study result - medication with significant interactions with trial medication.
- In case of smoking, start or cessation during the study.
- Pregnant or breastfeeding during the study period. Fertile women with a positive pregnancy test at the time of entry into the trial, or who do not use safe contraception during the project period.
Trial locations and contacts
1 site in 1 country.
Denmark1 site, 1 recruiting
- Recruiting
Copenhagen Neuromuscular Center, department of Neurology, Rigshospitalet
Copenhagen, DK-2100
Grete Andersen, MD, 004535456135, grete.andersen.01@regionh.dk
Grete Andersen, MD, Ph.D.
About this study
In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine.
According to the current corona pandemic, the investigators designed an app to use for data collection in the study. The app also ensures that patients who live far from the clinic more easily can participate.
Read the detailed description
On 18 May 2020, the National Board of Health in Denmark approved Namuscla for the treatment of myotonia. Myotonia is a congenital muscle disease that delays muscle relaxation after a muscle contraction. Myotonia can be disabling, but with treatment, most are able to work. Namuscla is the first registered drug to treat myotonia, however, the active part mexiletine is not new. It has been used off-label to treat myotonia for decades. The price for the new preparation is high with a daily cost (approx. 70 EU/dollar). In 2017, the investigators found evidence that treatment with lamotrigine is efficacious against myotonia. Treatment with lamotrigine can reduce the price to 150 EU/dollar per year.
Therefore, the aim of the study is to investigate whether there is a difference in the effects om myotonia and side effects of the two drugs, which can justify the significantly higher cost when treated with Namuscla in a double blinded, inferiority, cross-over study lasting 4 months.
According to the current corona pandemic, the study is designed without need of physical attendance so that inclusion and collection of experimental data can take place, by phone conversations, video-guided testing, and diary registration via a study-App. It also ensures that patients who live far away more easily can participate. Blood tests and ECGs can be performed locally.
- Conditions
- Non-Dystrophic Myotonia
- Being tested
- Lamotrigine, Namuscla
- Keywords
- Becker myotonia, Thomsen myotonia, Paramyotonia, Hyperkalemic periodic paralysis
- Medical subject headings
- Paralysis, Hyperkalemic Periodic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LamotrigineDrug
Lamotrigine tablets increasing dosis to maximal 300 mg daily
NamusclaDrug
Also called mexiletine
Namuscla tablets increasing dosis to maximal 501 mg daily
| Study group | Role | What they receive |
|---|---|---|
| Lamotrigine | Active comparator | An escalation phase of 28 days:
- tablet Lamotrigine 25 mg once daily in 14 days followed by 50 mg once daily in 14 days.
A treatment phase of 30 days:
- tablet Lamotrigine 100 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days. Receives: Drug: Lamotrigine |
| Namuscla | Active comparator | A placebo phase of 28 days:
- tablet placebo 25 mg once daily in 14 days followed by 50 mg once daily in 14 days.
A treatment phase of 30 days:
- tablet Namuscla 167 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days. Receives: Drug: Namuscla |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Myotonia Behavior Scale (MBS)
Each participant chose one of six statements about how myotonia affect their daily living. MBS is register once daily in a week. 1 is no symptoms of myotonia, 6 is invalidating symptoms of myotonia.
Measured without treatment and in the last week of treatment.
Measured: baseline and week 8
Secondary outcomes (6)
Change in Eye-myotonia
Eye opening test (time in seconds): Eyes are pinched with maximum strength for 5 sec, then opened ASAP. Repeated 5 times. Longer times measured more server myotonia in eye muscles.
Measured: baseline and week 8
Change in hand-myotonia
Hand opening test (time in seconds): Right hand is closed with maximum strength for five seconds before opened as soon as possible. Time is measured in seconds. Longer times measured more server myotonia in hand muscles. Repeated 5 times.
Measured: baseline and week 8
Change in time-up-and-go-test (TUG)
TUG - time-up\&go (time in seconds): After 10 minutes rest in a chair, participants get up, walk 3 meters, turn around, returning to the chair performed in regular pace. Longer times measured more server myotonia in legs muscles.
Measured: baseline and week 8
Change in Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
A questionnaire for patients with neuromuscular diseases measuring quality of life and the impact of the disease on everyday life. The INQoL is composed of 45 items investigating 4 dimensions, subdivided into 11 sub-dimensions. The scoring of the INQoL generates a profile (0-100). Higher score scores = higher impact of disorders. The form is translated in Danish.
Measured: baseline and week 8
Days with Side effects (SE)
Side effects are noted daily. Participants have three opportunities they can 1) cross no SE, 2) cross a specific SE on an alphabetically list containing all known SE for both drugs, or 3) they can describe a AE under other.
Measured: through study completion, in total 123 days
Change in Side Effect Scale (SE)
Each participant chose one of six statements about how side effects affect their daily living. SES is register once daily in a week. 1 is no side effects, 6 is invalidating side effects.
Measured: baseline and week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2022
- Study start5 Dec 2022
- Primary completion1 Apr 2024 (estimated)
- Study completion1 Apr 2024 (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.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Grete Andersen, MD
- Sponsor type
- University, hospital or other
- Collaborators
- GCP-Copenhagen, Region Capital Denmark, Danish Region, Lupin Atlantis Holdings S.A., ZiteLab
- Responsible party
- Sponsor-investigator, Grete Andersen, MD, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05639257
- Sponsor's protocol ID
- 2021-003784-94
- Status last verified
- Dec 2022
- First submitted
- 4 Oct 2022
- Last updated
- 6 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Myotonia - Lamotrigine Versus Namuscla
- ClinicalTrials.gov ID
- NCT05639257
- Acronym / sponsor ID
- 2021-003784-94
- Conditions
- Non-Dystrophic Myotonia
- Keywords
- Becker myotonia, Thomsen myotonia, Paramyotonia, Hyperkalemic periodic paralysis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Grete Andersen, MDUniversity, hospital or other
- Collaborators
- GCP-Copenhagen, Region Capital Denmark, Danish Region, Lupin Atlantis Holdings S.A., ZiteLab
- Study start
- 5 Dec 2022 (actual)
- Primary completion
- 1 Apr 2024 (estimated)
- Study completion
- 1 Apr 2024 (estimated)
- First posted
- 6 Dec 2022 (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.
In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine.
According to the current corona pandemic, the investigators designed an app to use for data collection in the study. The app also ensures that patients who live far from the clinic more easily can participate.
Detailed description
On 18 May 2020, the National Board of Health in Denmark approved Namuscla for the treatment of myotonia. Myotonia is a congenital muscle disease that delays muscle relaxation after a muscle contraction. Myotonia can be disabling, but with treatment, most are able to work. Namuscla is the first registered drug to treat myotonia, however, the active part mexiletine is not new. It has been used off-label to treat myotonia for decades. The price for the new preparation is high with a daily cost (approx. 70 EU/dollar). In 2017, the investigators found evidence that treatment with lamotrigine is efficacious against myotonia. Treatment with lamotrigine can reduce the price to 150 EU/dollar per year.
Therefore, the aim of the study is to investigate whether there is a difference in the effects om myotonia and side effects of the two drugs, which can justify the significantly higher cost when treated with Namuscla in a double blinded, inferiority, cross-over study lasting 4 months.
According to the current corona pandemic, the study is designed without need of physical attendance so that inclusion and collection of experimental data can take place, by phone conversations, video-guided testing, and diary registration via a study-App. It also ensures that patients who live far away more easily can participate. Blood tests and ECGs can be performed locally.
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 |
|---|---|---|
| Lamotrigine | Active comparator | Drug: LamotrigineAn escalation phase of 28 days: - tablet Lamotrigine 25 mg once daily in 14 days followed by 50 mg once daily in 14 days. A treatment phase of 30 days: - tablet Lamotrigine 100 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days. |
| Namuscla | Active comparator | Drug: NamusclaA placebo phase of 28 days: - tablet placebo 25 mg once daily in 14 days followed by 50 mg once daily in 14 days. A treatment phase of 30 days: - tablet Namuscla 167 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Capable adult women and men (age ≥ 18 years).
- Diagnosed with Myotonia Congenita (Becker and Thomsen type), Paramyotonia Congenita, or Hyperkalemic periodic paralysis.
- Myotonia under treatment or which significantly limits the daily activities (MBS> 2).
Key exclusion criteria (5 listed)
- Allergy to lamotrigine, mexiletine, or the inactive ingredients in trial medication.
- Disease, which is affected by trial medication such as heart disease (ischemia and arrhythmia), epilepsy, and significant renal or hepatic failure.
- Treatment that, in the opinion of the project manager, can affect the study result - medication with significant interactions with trial medication.
- In case of smoking, start or cessation during the study.
- Pregnant or breastfeeding during the study period. Fertile women with a positive pregnancy test at the time of entry into the trial, or who do not use safe contraception during the project period.
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 |
|---|---|---|
| LamotrigineLamotrigine tablets increasing dosis to maximal 300 mg daily | Drug | Lamotrigine |
| NamusclaAlso: mexiletineNamuscla tablets increasing dosis to maximal 501 mg daily | Drug | Namuscla |
Primary outcome
Change in Myotonia Behavior Scale (MBS)
Time frame: baseline and week 8
Each participant chose one of six statements about how myotonia affect their daily living. MBS is register once daily in a week. 1 is no symptoms of myotonia, 6 is invalidating symptoms of myotonia. Measured without treatment and in the last week of treatment.
6 secondary outcomes
Change in Eye-myotonia
Time frame: baseline and week 8
Change in hand-myotonia
Time frame: baseline and week 8
Change in time-up-and-go-test (TUG)
Time frame: baseline and week 8
Change in Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
Time frame: baseline and week 8
Days with Side effects (SE)
Time frame: through study completion, in total 123 days
Change in Side Effect Scale (SE)
Time frame: baseline and week 8
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2022.
- Individual participant dataSharing planned: No
Related trials for Non-Dystrophic Myotonia
RecruitingPhase 3
An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR
Myotonia
Not yet recruiting
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lamotrigine and Namuscla in people with Non-Dystrophic Myotonia, run by Grete Andersen, MD at 1 site in Denmark.
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 5 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: status not confirmed, as of the sponsor's last update on 6 Dec 2022. 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 Denmark. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Grete Andersen (35453545, grete.andersen.01@regionh.dk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Myotonia Behavior Scale (MBS) (time frame: baseline and week 8). 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 6 Dec 2022.