Pompe Disease QMUS and EIM

September 24, 2020 updated by: Duke University

Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

25

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Device Feasibility
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: QMUS
All patients will undergo serial quantitative muscle ultrasound.
Device: Muscle ultrasound
Ultrasound images of limb muscles will be collected.
Other: EIM
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percent change in muscle ultrasound echointensity as compared to baseline
Time Frame: Baseline, 1-2 years
Baseline, 1-2 years
Percent change in muscle electrical impedance myography as compared to baseline
Time Frame: Baseline, 1-2 years
Baseline, 1-2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures
Time Frame: 1-2 years
1-2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Duke University

Collaborators

Genzyme, a Sanofi Company

Investigators

  • Principal Investigator: Lisa D Hobson-Webb, MD, Duke University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 24, 2016

Primary Completion (Actual)

July 31, 2020

Study Completion (Actual)

July 31, 2020

Study Registration Dates

First Submitted

April 11, 2016

First Submitted That Met QC Criteria

April 13, 2016

First Posted (Estimate)

April 19, 2016

Study Record Updates

Last Update Posted (Actual)

September 25, 2020

Last Update Submitted That Met QC Criteria

September 24, 2020

Last Verified

February 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00072199

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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