Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 9 of 103 for:    Pompe Disease

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 29, 2019
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Duke University

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.

Condition or disease Intervention/treatment Phase
Pompe Disease Device: Muscle ultrasound Other: Electrical Impedance Myography Not Applicable

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Device Feasibility
Official Title: Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Actual Study Start Date : October 24, 2016
Estimated Primary Completion Date : April 20, 2020
Estimated Study Completion Date : April 20, 2020


Arm Intervention/treatment
QMUS
All patients will undergo serial quantitative muscle ultrasound.
Device: Muscle ultrasound
Ultrasound images of limb muscles will be collected.

EIM
All patients will undergo serial electrical impedance myography measures.
Other: Electrical Impedance Myography
A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.




Primary Outcome Measures :
  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 Outcome Measures :
  1. Inter-rater reliability of muscle ultrasound and electrical impedance myography measures [ Time Frame: 1-2 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pompe disease.

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02742298


Locations
Layout table for location information
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
Genzyme, a Sanofi Company
Investigators
Layout table for investigator information
Principal Investigator: Lisa D Hobson-Webb, MD Duke University

Layout table for additonal information
Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT02742298     History of Changes
Other Study ID Numbers: Pro00072199
First Posted: April 19, 2016    Key Record Dates
Last Update Posted: March 29, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Duke University:
Neuromuscular Ultrasound
Electrical Impedance Myography

Additional relevant MeSH terms:
Layout table for MeSH terms
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors