NCT02251457

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Aug 2014

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2014

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

September 25, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 29, 2014

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 18, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 18, 2017

Completed
Last Updated

March 5, 2019

Status Verified

March 1, 2019

Enrollment Period

3.4 years

First QC Date

September 25, 2014

Last Update Submit

March 1, 2019

Conditions

Myotonia CongenitaParamyotonia CongenitaMyotonic Dystrophy 1

Keywords

Myotonia CongenitaranolazineOhio StateOpen LabelParamyotonia CongenitaMyotonic Dystrophy 1

Outcome Measures

Primary Outcomes (3)

  • 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

    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

    1 month

  • Electromyography (EMG) Myotonia

    To see if the electrical potentials produced by the muscle fibers change.

    1 month

Secondary Outcomes (1)

  • Electrocardiogram (ECG)

    1 month

Study Arms (1)

ranolazine

EXPERIMENTAL

ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks

Drug: Ranolazine

Interventions

RanolazineDRUG

Ranexa is FDA approved for chronic angina

Also known as: Ranexa®
ranolazine

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43221, United States

Location

Related Publications (3)

  • 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.

    PMID: 25515836BACKGROUND

  • Spillane 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.

    PMID: 40197813DERIVED

  • Lorusso 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.

    PMID: 30390395DERIVED

MeSH Terms

Conditions

Myotonia CongenitaMyotonic DisordersMyotonic Dystrophy

Interventions

Ranolazine

Condition Hierarchy (Ancestors)

Muscular DiseasesMusculoskeletal DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMuscular DystrophiesMuscular Disorders, Atrophic

Intervention Hierarchy (Ancestors)

AcetanilidesAnilidesAmidesOrganic ChemicalsAniline CompoundsAminesPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • William D Arnold, MD

    Ohio State University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

September 25, 2014

First Posted

September 29, 2014

Study Start

August 1, 2014

Primary Completion

December 18, 2017

Study Completion

December 18, 2017

Last Updated

March 5, 2019

Record last verified: 2019-03

Locations

US(1)