Characteristics of Nondystrophic Myotonias

NCT ID: NCT00244413

Last Updated: 2013-03-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

94 participants

Study Classification

OBSERVATIONAL

Study Start Date

2006-02-28

Study Completion Date

2012-09-30

Brief Summary

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Nondystrophic myotonias (NDM) are muscle disorders caused by genetic abnormalities in certain muscle cell membrane proteins. Individuals with NDM experience limited muscle relaxation, which causes pain, weakness, and impaired physical activity. The purpose of this study is to better characterize the clinical features and symptoms of NDM.

Detailed Description

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Nondystrophic myotonias are muscle disorders caused by abnormal muscle cell membrane proteins that affect the control of muscle fiber contraction. These disorders are extremely rare, and little is known about how to best treat the various subtypes of NDM. The purpose of this study is to characterize the clinical features and symptoms of NDM as well as to pair this data with specific NDM subtypes. In turn, this may lead to the development of improved treatments. The study will also establish clinical endpoints for use in future studies.

This multi-center observational study will involve both a cross-sectional data analysis and a prospective longitudinal analysis. Participants will initially attend a one-day outpatient study visit. Various baseline measurements will be collected, including demographics, medical history, and quality of life measures. Blood samples will be taken to evaluate laboratory values and genetic factors. Participants will undergo manual muscle testing (MMT), clinical myotonia assessments, and functional movement assessments. Routine nerve conduction studies and electromyography (EMG) will also be performed in order to test for the presence of myotonia in specific muscles. Annual follow-up evaluations will occur 1 and 2 years following the first study visit.

Conditions

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Nondystrophic Myotonias Myotonia Congenita Myotonic Disorders

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Clinical symptoms or signs suggestive of myotonia
* Presence of myotonic potentials on electromyography (EMG)
* Persistence of symptoms and signs after discontinuation of medications that produce myotonia; such medications include fibric acid derivatives, hydroxymethylglutaryl CoA reductase inhibitors, chloroquine, and colchicine
* Absence of features suggestive of myotonic dystrophy, including ptosis, temporal wasting, mandibular weakness, cataracts occurring before age 50, and evidence of multisystem defects (cardiac conduction defects, hypogonadism)

Exclusion Criteria

* Any other neurologic condition that might affect the assessment of the study measurements
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Office of Rare Diseases (ORD)

NIH

Sponsor Role collaborator

Rare Diseases Clinical Research Network

NETWORK

Sponsor Role collaborator

Richard Barohn, MD

OTHER

Sponsor Role lead

Responsible Party

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Richard Barohn, MD

Gertrude and Dewey Zeigler Professor of Neurology and Chair

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Richard Barohn, MD

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Locations

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University of Kansas Medical Center, Department of Neurology

Kansas City, Kansas, United States

Site Status

Brigham & Women's Hospital, Department of Neurology

Boston, Massachusetts, United States

Site Status

University of Rochester School of Medicine and Dentistry, Department of Neurology

Rochester, New York, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

London Health Sciences Centre, University Hospital

London, Ontario, Canada

Site Status

Center for Neuromuscular Disease, Institute of Neurology and National Hospital for Neurology

London, , United Kingdom

Site Status

Countries

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United States Canada United Kingdom

References

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Torbergsen T, Hodnebo A, Brautaset NJ, Loseth S, Stalberg E. A rare form of painful nondystrophic myotonia. Clin Neurophysiol. 2003 Dec;114(12):2347-54. doi: 10.1016/s1388-2457(03)00275-x.

Reference Type BACKGROUND
PMID: 14652094 (View on PubMed)

Renner DR, Ptacek LJ. Periodic paralyses and nondystrophic myotonias. Adv Neurol. 2002;88:235-52. No abstract available.

Reference Type BACKGROUND
PMID: 11908229 (View on PubMed)

Cannon SC. Spectrum of sodium channel disturbances in the nondystrophic myotonias and periodic paralyses. Kidney Int. 2000 Mar;57(3):772-9. doi: 10.1046/j.1523-1755.2000.00914.x.

Reference Type BACKGROUND
PMID: 10720928 (View on PubMed)

Cannon SC. From mutation to myotonia in sodium channel disorders. Neuromuscul Disord. 1997 Jun;7(4):241-9. doi: 10.1016/s0960-8966(97)00430-6.

Reference Type BACKGROUND
PMID: 9196906 (View on PubMed)

Moxley RT 3rd. The myotonias: their diagnosis and treatment. Compr Ther. 1996 Jan;22(1):8-21. No abstract available.

Reference Type BACKGROUND
PMID: 8654027 (View on PubMed)

Brown RH Jr. Ion channel mutations in periodic paralysis and related myotonic diseases. Ann N Y Acad Sci. 1993 Dec 20;707:305-16. doi: 10.1111/j.1749-6632.1993.tb38061.x. No abstract available.

Reference Type BACKGROUND
PMID: 9137561 (View on PubMed)

Other Identifiers

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10263

Identifier Type: -

Identifier Source: org_study_id

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