Myotonia CongenitaPhase 1NCT02251457
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
A Phase 1 study testing Ranolazine in people with Myotonia Congenita and Paramyotonia Congenita, run by Ohio State University at 1 site in United States.
Trial summary
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- What is being tested?
- Ranolazine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2014to 18 Dec 2017
- Phase
- Phase 1
- 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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1 established by genetic testing in the subject or in a first-degree relative.
- Clinically evident myotonia
Who cannot join
- Contraindications to ranolazine use:
- for fungus infection: ketoconazole (Nizoral), itraconazole (Sporanox, Onmel)
- for infection: clarithromycin (Biaxin)
- for depression: nefazodone
- for HIV: nelfinavir (Viracept), ritonavir (Norvir), lopinavir and ritonavir (Kaletra), indinavir (Crixivan), saquinavir (Invirase).
- for tuberculosis (TB): rifampin (Rifadin), rifabutin (Mycobutin), rifapentine (Priftin)
Show all 13Show fewer
- for seizures: phenobarbital, phenytoin (Phenytek, Dilantin, Dilantin-125), carbamazepine (Tegretol)
- the herbal supplement St. John's wort
- you have scarring (cirrhosis) of your liver
- Concurrent use of mexiletine, lacosamide, acetazolamide, phenytoin, quinine, procainamide, Saint John wort or tocainide. Patients who were previously treated with these medications may participate. They need to be off of the medication for at least a week prior to enrollment.
- QTc >470 ms for men and >480 ms for women.
- Women who are pregnant or breastfeeding
- Direct family history of sudden cardiac death
Trial locations and contacts
1 site in 1 country.
United States1 site
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43221
About this study
Official title: Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks.
Read the detailed description
Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. The data from this animal model suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that participate will be placed on active drug. The investigators want to see if this drug is safe to take without causing too many side effects for people with myotonia congenita, paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State University for study visits. Participants will take ranolazine for four weeks. Participants can expect a total of 4 study visits and 2 phone calls over the 5 week period.
- Being tested
- Ranolazine
- Keywords
- Myotonia Congenita, ranolazine, Ohio State, Open Label, Paramyotonia Congenita, Myotonic Dystrophy 1
- Medical subject headings
- Myotonia Congenita, Myotonic Disorders, Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RanolazineDrug
Also called Ranexa®
Ranexa is FDA approved for chronic angina
| Study group | Role | What they receive |
|---|---|---|
| ranolazine | Experimental | ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks Receives: Drug: Ranolazine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
quality of life measurements for overall health and neuromuscular disease
Measured: 1 month
Muscle tasks
The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
Measured: 1 month
Electromyography (EMG) Myotonia
To see if the electrical potentials produced by the muscle fibers change.
Measured: 1 month
Secondary outcomes (1)
Electrocardiogram (ECG)
to measure heart function and observe QT interval (a measure of the time between the start of the Q wave and the end of the T wave in the heart's electrical cycle)
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2014 (estimated)
- Study startAug 2014
- Primary completion18 Dec 2017
- Study completion18 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Myotonia CongenitaStudies of the same condition, open and under way.
- More trials run by Ohio State UniversityThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ohio State University
- Sponsor type
- University, hospital or other
- Collaborators
- Gilead Sciences
- Responsible party
- Principal investigator, William Arnold, Ohio State University
- Investigators
- William D Arnold, MD, Principal investigator, Ohio State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02251457
- Sponsor's protocol ID
- IN-US-259-1605
- Other IDs
- IN-US-259-1605
- Status last verified
- Mar 2019
- First submitted
- 25 Sep 2014
- Last updated
- 5 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Publications
Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9.
PubMed 25515836Spillane J, Trip J, Drost G, Faber CG, Hanna MG, Nevitt SJ, Vivekanandam V. Drug treatment for myotonia. Cochrane Database Syst Rev. 2025 Apr 8;4(4):CD004762. doi: 10.1002/14651858.CD004762.pub3.
PubMed 40197813Lorusso S, Kline D, Bartlett A, Freimer M, Agriesti J, Hawash AA, Rich MM, Kissel JT, David Arnold W. Open-label trial of ranolazine for the treatment of paramyotonia congenita. Muscle Nerve. 2019 Feb;59(2):240-243. doi: 10.1002/mus.26372. Epub 2018 Dec 21.
PubMed 30390395
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT02251457
- Acronym / sponsor ID
- IN-US-259-1605
- Keywords
- Myotonia Congenita, ranolazine, Ohio State, Open Label, Paramyotonia Congenita, Myotonic Dystrophy 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Ohio State UniversityUniversity, hospital or other
- Collaborators
- Gilead Sciences
- Study start
- Aug 2014
- Primary completion
- 18 Dec 2017 (actual)
- Study completion
- 18 Dec 2017 (actual)
- First posted
- 29 Sep 2014 (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.
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks.
Detailed description
Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. The data from this animal model suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that participate will be placed on active drug. The investigators want to see if this drug is safe to take without causing too many side effects for people with myotonia congenita, paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State University for study visits. Participants will take ranolazine for four weeks. Participants can expect a total of 4 study visits and 2 phone calls over the 5 week period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ranolazine | Experimental | Drug: Ranolazineranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1 established by genetic testing in the subject or in a first-degree relative.
- Clinically evident myotonia
Key exclusion criteria (13 listed)
- Contraindications to ranolazine use:
- Concurrent use of mexiletine, lacosamide, acetazolamide, phenytoin, quinine, procainamide, Saint John wort or tocainide. Patients who were previously treated with these medications may participate. They need to be off of the medication for at least a week prior to enrollment.
- QTc >470 ms for men and >480 ms for women.
- Women who are pregnant or breastfeeding
- Direct family history of sudden cardiac death
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 |
|---|---|---|
| RanolazineAlso: Ranexa®Ranexa is FDA approved for chronic angina | Drug | ranolazine |
Primary outcome
Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
Time frame: 1 month
quality of life measurements for overall health and neuromuscular disease
Muscle tasks
Time frame: 1 month
The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
Electromyography (EMG) Myotonia
Time frame: 1 month
To see if the electrical potentials produced by the muscle fibers change.
1 secondary outcome
Electrocardiogram (ECG)
Time frame: 1 month
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 5 Mar 2019.
- Background: PubMed 25515836Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9.
- Derived from this study: PubMed 40197813Spillane J, Trip J, Drost G, Faber CG, Hanna MG, Nevitt SJ, Vivekanandam V. Drug treatment for myotonia. Cochrane Database Syst Rev. 2025 Apr 8;4(4):CD004762. doi: 10.1002/14651858.CD004762.pub3.
- Derived from this study: PubMed 30390395Lorusso S, Kline D, Bartlett A, Freimer M, Agriesti J, Hawash AA, Rich MM, Kissel JT, David Arnold W. Open-label trial of ranolazine for the treatment of paramyotonia congenita. Muscle Nerve. 2019 Feb;59(2):240-243. doi: 10.1002/mus.26372. Epub 2018 Dec 21.
- Individual participant dataSharing planned: Undecided
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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.
William D Arnold, MD principal investigator
Ohio State University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ranolazine in people with Myotonia Congenita and Paramyotonia Congenita, run by Ohio State University at 1 site in United States.
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 13 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 5 Mar 2019. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL) (time frame: 1 month). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02251457, which also lists 3 linked publications.
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 5 Mar 2019.