Pompe Disease QMUS and EIM

Not Applicable

Completed

• Conditions: Pompe Disease
• Interventions: Device: Muscle ultrasound; Other: Electrical Impedance Myography

• Registration Number: NCT02742298
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-04-11
• Study First Submitted QC: 2016-04-13
• Study First Posted: 2016-04-19
• Study Start: 2016-10-24
• Status Verified: 2020-02
• Primary Completion: 2020-07-31
• Study Completion: 2020-07-31
• Last Update Submitted: 2020-09-24
• Last Update Posted: 2020-09-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

• Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
QMUS	Muscle ultrasound	All patients will undergo serial quantitative muscle ultrasound.
EIM	Electrical Impedance Myography	All patients will undergo serial electrical impedance myography measures.

Primary Outcome Measures

Name	Time	Method
Percent change in muscle ultrasound echointensity as compared to baseline	Baseline, 1-2 years
Percent change in muscle electrical impedance myography as compared to baseline	Baseline, 1-2 years

Secondary Outcome Measures

Name	Time	Method
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures	1-2 years

Trial Locations

Locations (1)

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States