Pompe Disease QMUS and EIM

NCT ID: NCT02742298

Last Updated: 2020-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-24

Study Completion Date

2020-07-31

Brief Summary

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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

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Conditions

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Pompe Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

DEVICE_FEASIBILITY

Blinding Strategy

SINGLE

Investigators

Study Groups

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QMUS

All patients will undergo serial quantitative muscle ultrasound.

Group Type OTHER

Muscle ultrasound

Intervention Type DEVICE

Ultrasound images of limb muscles will be collected.

EIM

All patients will undergo serial electrical impedance myography measures.

Group Type OTHER

Electrical Impedance Myography

Intervention Type OTHER

A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.

Interventions

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Muscle ultrasound

Ultrasound images of limb muscles will be collected.

Intervention Type DEVICE

Electrical Impedance Myography

A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria

* Age \<18 years
* Inability to provide informed consent.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role collaborator

Duke University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lisa D Hobson-Webb, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

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Duke University Medical Center

Durham, North Carolina, United States

Site Status

Countries

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United States

Other Identifiers

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Pro00072199

Identifier Type: -

Identifier Source: org_study_id

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