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Pompe Disease QMUS and EIM

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02742298
Recruitment Status : Completed
First Posted : April 19, 2016
Last Update Posted : September 25, 2020
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Duke University

Brief Summary:

Study Objectives:

  1. Determine the correlation between quantitative muscle ultrasound (QMUS), electrical impedance myography (EIM) and currently accepted measures of physical function.
  2. Determine the reliability of EIM measures performed in the home through use of a hand held device.
  3. Determine if QMUS and EIM can detect pre-clinical changes in Pompe disease.

Condition or disease Intervention/treatment Phase
Pompe Disease Device: Muscle ultrasound Other: Electrical Impedance Myography Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Device Feasibility
Official Title: Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Actual Study Start Date : October 24, 2016
Actual Primary Completion Date : July 31, 2020
Actual Study Completion Date : July 31, 2020

Arm Intervention/treatment
All patients will undergo serial quantitative muscle ultrasound.
Device: Muscle ultrasound
Ultrasound images of limb muscles will be collected.

All patients will undergo serial electrical impedance myography measures.
Other: Electrical Impedance Myography
A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.

Primary Outcome Measures :
  1. Percent change in muscle ultrasound echointensity as compared to baseline [ Time Frame: Baseline, 1-2 years ]
  2. Percent change in muscle electrical impedance myography as compared to baseline [ Time Frame: Baseline, 1-2 years ]

Secondary Outcome Measures :
  1. Inter-rater reliability of muscle ultrasound and electrical impedance myography measures [ Time Frame: 1-2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

  • Age <18 years
  • Inability to provide informed consent.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02742298

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United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
Genzyme, a Sanofi Company
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Principal Investigator: Lisa D Hobson-Webb, MD Duke University
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Responsible Party: Duke University Identifier: NCT02742298    
Other Study ID Numbers: Pro00072199
First Posted: April 19, 2016    Key Record Dates
Last Update Posted: September 25, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Duke University:
Neuromuscular Ultrasound
Electrical Impedance Myography
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors