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Pompe Disease QMUS and EIM

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02742298
Recruitment Status : Active, not recruiting
First Posted : April 19, 2016
Last Update Posted : March 2, 2020
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Duke University

Tracking Information
First Submitted Date  ICMJE April 11, 2016
First Posted Date  ICMJE April 19, 2016
Last Update Posted Date March 2, 2020
Actual Study Start Date  ICMJE October 24, 2016
Estimated Primary Completion Date April 20, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 13, 2016)
  • Percent change in muscle ultrasound echointensity as compared to baseline [ Time Frame: Baseline, 1-2 years ]
  • Percent change in muscle electrical impedance myography as compared to baseline [ Time Frame: Baseline, 1-2 years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 13, 2016)
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures [ Time Frame: 1-2 years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pompe Disease QMUS and EIM
Official Title  ICMJE Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Brief Summary

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 Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Device Feasibility
Condition  ICMJE Pompe Disease
Intervention  ICMJE
  • Device: Muscle ultrasound
    Ultrasound images of limb muscles will be collected.
  • Other: Electrical Impedance Myography
    A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.
Study Arms  ICMJE
  • QMUS
    All patients will undergo serial quantitative muscle ultrasound.
    Intervention: Device: Muscle ultrasound
  • EIM
    All patients will undergo serial electrical impedance myography measures.
    Intervention: Other: Electrical Impedance Myography
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: April 13, 2016)
25
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 20, 2020
Estimated Primary Completion Date April 20, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

  • Age <18 years
  • Inability to provide informed consent.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02742298
Other Study ID Numbers  ICMJE Pro00072199
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Duke University
Study Sponsor  ICMJE Duke University
Collaborators  ICMJE Genzyme, a Sanofi Company
Investigators  ICMJE
Principal Investigator: Lisa D Hobson-Webb, MD Duke University
PRS Account Duke University
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP