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Pompe Disease QMUS and EIM

Primary Purpose

Pompe Disease

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Muscle ultrasound
Electrical Impedance Myography
Sponsored by
Duke University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional device feasibility trial for Pompe Disease focused on measuring Neuromuscular Ultrasound, Electrical Impedance Myography

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

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

Sites / Locations

  • Duke University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Other

Arm Label

QMUS

EIM

Arm Description

All patients will undergo serial quantitative muscle ultrasound.

All patients will undergo serial electrical impedance myography measures.

Outcomes

Primary Outcome Measures

Percent change in muscle ultrasound echointensity as compared to baseline
Percent change in muscle electrical impedance myography as compared to baseline

Secondary Outcome Measures

Inter-rater reliability of muscle ultrasound and electrical impedance myography measures

Full Information

First Posted
April 11, 2016
Last Updated
September 24, 2020
Sponsor
Duke University
Collaborators
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT02742298
Brief Title
Pompe Disease QMUS and EIM
Official Title
Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Study Type
Interventional

2. Study Status

Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
October 24, 2016 (Actual)
Primary Completion Date
July 31, 2020 (Actual)
Study Completion Date
July 31, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Duke University
Collaborators
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease
Keywords
Neuromuscular Ultrasound, Electrical Impedance Myography

7. Study Design

Primary Purpose
Device Feasibility
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
Investigator
Allocation
Non-Randomized
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
QMUS
Arm Type
Other
Arm Description
All patients will undergo serial quantitative muscle ultrasound.
Arm Title
EIM
Arm Type
Other
Arm Description
All patients will undergo serial electrical impedance myography measures.
Intervention Type
Device
Intervention Name(s)
Muscle ultrasound
Intervention Description
Ultrasound images of limb muscles will be collected.
Intervention Type
Other
Intervention Name(s)
Electrical Impedance Myography
Intervention Description
A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.
Primary Outcome Measure Information:
Title
Percent change in muscle ultrasound echointensity as compared to baseline
Time Frame
Baseline, 1-2 years
Title
Percent change in muscle electrical impedance myography as compared to baseline
Time Frame
Baseline, 1-2 years
Secondary Outcome Measure Information:
Title
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures
Time Frame
1-2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Genetically confirmed diagnosis of Pompe disease. Exclusion Criteria: Age <18 years Inability to provide informed consent.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lisa D Hobson-Webb, MD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Pompe Disease QMUS and EIM

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