Electrical Impedance Myography as an Outcome Measure in Amyotrophic Lateral Sclerosis Clinical Trials

NCT00620698 | Completed Results

• 1 other identifier: EIMALS
• Study Type: observational
• Target: 89
• Locations: 1 country
• Sites: 8

Brief Summary

Trials evaluating new therapies for stopping or slowing the progression of ALS depend critically upon the use of outcome measures to assess whether a potential treatment is effective. The more effective an outcome measure, the fewer patients need to be enrolled and the shorter the trial. Many outcome measures have been used over the years, including strength assessments, breathing tests, functional status surveys, and nerve testing, but all are far from ideal. A new method, called electrical impedance myography (EIM) appears to be especially promising in that it provides very consistent data from one testing session to the next, is sensitive to the muscle deterioration that occurs in ALS, and is entirely painless and non-invasive. In this study, investigators from multiple institutions plan to compare several different outcome measures, including EIM, in approximately 120 ALS patients, with each patient being followed for a period of one year. All of these measures will be compared to one another and an assessment of their ability to detect disease progression made. Our goal will be to determine whether EIM can serve as a valuable new outcome measure, ultimately leading to substantially faster, more effective ALS trials requiring fewer patients.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

amyotrophic lateral sclerosis; motor neuron disease; outcome measure; biomarker; impedance

Outcome Measures

Primary Outcomes (1)

• Electrical Impedance Myography

  The main outcome measure was the coefficient of variation (CoV) in the rate of the decline for each measure over time. The CoV was calculated by dividing the standard deviation in the rate of decline across the group of subjects and dividing that by the mean rate of decline for the cohort. This approach was taken for each of the measures being evaluated (ALS Functional Rating Scale-Revised, Handheld dynamometry, Electrical impedance myography). The lower the CoV in the rate of decline, the more sensitive it is to identifying a potential treatment effect, since it suggests gives a measure of homogeneity of the rate of decline across the population as well as the overall rate of decline. The smaller the standard deviation across the group and the larger the mean rate of decline across the group, the lower the CoV and the fewer number of subjects needed for a potential clinical trial using that outcome measure.

  6 months

Secondary Outcomes (2)

• ALS Functional Rating Scale

  6 months

• Handheld Dynamometry

  6 months

Study Arms (1)

ALS patients

Patients with clinically established amyotrophic lateral sclerosis

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients with amyotrophic lateral sclerosis (ALS)

You may qualify if:

• Definite or probably ALS by El Escorial criteria
• Muscle strength of at 3.5 in one limb

You may not qualify if:

• Forced vital capacity of less than 70%
• Atypical forms of motor neuron disease (monomelic amyotrophy, primary lateral sclerosis)
• Pacemaker

Sponsors & Collaborators

• Beth Israel Deaconess Medical Center: lead
• ALS Association: collaborator

Study Sites (8)

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Emory University

Atlanta, Georgia, United States

Location

Johns Hopkins

Baltimore, Maryland, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02446, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, United States

Location

Upstate Medical Center

Syracuse, New York, United States

Location

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

University of Virginia Medical Center

Charlottesville, Virginia, 22908, United States

Location

Related Publications (1)

• Rutkove SB, Zhang H, Schoenfeld DA, Raynor EM, Shefner JM, Cudkowicz ME, Chin AB, Aaron R, Shiffman CA. Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials. Clin Neurophysiol. 2007 Nov;118(11):2413-8. doi: 10.1016/j.clinph.2007.08.004. Epub 2007 Sep 25.

  PMID: 17897874 BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Seward Rutkove
• Organization: Beth Israel Deaconess Medical Center

srutkove@bidmc.harvard.edu

617-667-8130

Study Officials

• Seward B Rutkove, MD

  Beth Israel Deaconess Medical Center

  PRINCIPAL INVESTIGATOR

• Jeremy M Shefner, MD, PhD

  Upstate Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Invesigator

Study Record Dates

• First Submitted: February 9, 2008
• First Posted: February 21, 2008
• Study Start: May 1, 2007
• Primary Completion: March 1, 2011
• Study Completion: March 1, 2012
• Last Updated: September 25, 2014
• Results First Posted: September 10, 2014

Record last verified: 2014-09

Locations

US (8)