NCT02742298

Brief Summary

Study Objectives:

  1. 1.Determine the correlation between quantitative muscle ultrasound (QMUS), electrical impedance myography (EIM) and currently accepted measures of physical function.
  2. 2.Determine the reliability of EIM measures performed in the home through use of a hand held device.
  3. 3.Determine if QMUS and EIM can detect pre-clinical changes in Pompe disease.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Oct 2016

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 11, 2016

Completed
8 days until next milestone

First Posted

Study publicly available on registry

April 19, 2016

Completed
6 months until next milestone

Study Start

First participant enrolled

October 24, 2016

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2020

Completed
Last Updated

September 25, 2020

Status Verified

February 1, 2020

Enrollment Period

3.8 years

First QC Date

April 11, 2016

Last Update Submit

September 24, 2020

Conditions

Pompe Disease

Keywords

Neuromuscular UltrasoundElectrical Impedance Myography

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (1)

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

    1-2 years

Study Arms (2)

QMUS

OTHER

All patients will undergo serial quantitative muscle ultrasound.

Device: Muscle ultrasound

EIM

OTHER

All patients will undergo serial electrical impedance myography measures.

Other: Electrical Impedance Myography

Interventions

Muscle ultrasoundDEVICE

Ultrasound images of limb muscles will be collected.

QMUS
Electrical Impedance MyographyOTHER

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

EIM

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Genetically confirmed diagnosis of Pompe disease.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Lisa D Hobson-Webb, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
INVESTIGATOR
Purpose
DEVICE FEASIBILITY
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 11, 2016

First Posted

April 19, 2016

Study Start

October 24, 2016

Primary Completion

July 31, 2020

Study Completion

July 31, 2020

Last Updated

September 25, 2020

Record last verified: 2020-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)