Pompe Disease QMUS and EIM

Duke University

Status: Completed

Conditions: Pompe Disease

Treatments: Device: Muscle ultrasound; Other: Electrical Impedance Myography

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02742298

Pro00072199

Details and patient eligibility

About

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.

Enrollment: 25 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Genetically confirmed diagnosis of Pompe disease.

Exclusion criteria

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

Trial design

Primary purpose: Device Feasibility

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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25 participants in 2 patient groups

QMUS

Other group

Description:

All patients will undergo serial quantitative muscle ultrasound.

Treatment:

Device: Muscle ultrasound

EIM

Other group

Description:

All patients will undergo serial electrical impedance myography measures.

Treatment:

Other: Electrical Impedance Myography