Pompe Disease QMUS and EIM

Sponsor

Duke University (Other)

Overall Status

Completed

CT.gov ID

NCT02742298

Collaborator

Genzyme, a Sanofi Company (Industry)

Enrollment

Location

Arms

45.2

Actual Duration (Months)

0.6

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Pompe Disease	Device: Muscle ultrasound Other: Electrical Impedance Myography	N/A

Study Design

Study Type:

Interventional

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 :

Oct 24, 2016

Actual Primary Completion Date :

Jul 31, 2020

Actual Study Completion Date :

Jul 31, 2020

Arms and Interventions

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.

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.

Outcome Measures

Primary Outcome Measures

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

Inter-rater reliability of muscle ultrasound and electrical impedance myography measures [1-2 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

Age <18 years
Inability to provide informed consent.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Duke University Medical Center	Durham	North Carolina	United States	27710

Sponsors and Collaborators

Duke University
Genzyme, a Sanofi Company

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Duke University

ClinicalTrials.gov Identifier:

NCT02742298

Other Study ID Numbers:

Pro00072199

First Posted:

Apr 19, 2016

Last Update Posted:

Sep 25, 2020

Last Verified:

Feb 1, 2020

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Keywords provided by Duke University

Neuromuscular Ultrasound

Electrical Impedance Myography

Additional relevant MeSH terms:

Glycogen Storage Disease Type II

Study Results

No Results Posted as of Sep 25, 2020