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OpenTrials
Completed

NCT Number: NCT02742298

Pompe Disease QMUS and EIM

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.

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Key information

Conditions

Pompe Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion criteria

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

Treatment and study plan

Muscle ultrasound

Device

Ultrasound images of limb muscles will be collected.

Electrical Impedance Myography

Other

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

Primary outcomes

  1. Percent change in muscle ultrasound echointensity as compared to baseline

    Time frame: Baseline, 1-2 years

  2. Percent change in muscle electrical impedance myography as compared to baseline

    Time frame: Baseline, 1-2 years

Secondary outcomes

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

    Time frame: 1-2 years

Sponsors and collaborators

Lead sponsor

Duke University

Other

Collaborators

  • Genzyme, a Sanofi Company

Registry information

Official study title

Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease

Important dates

Study start
2016
Primary completion
2020
Study completion
2020
First posted
Apr 19, 2016
Registry last updated
Sep 25, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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