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Identification of Tongue Involvement in Late-Onset Pompe Disease

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ClinicalTrials.gov Identifier: NCT02765828
Recruitment Status : Recruiting
First Posted : May 9, 2016
Last Update Posted : March 31, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

April 22, 2016
May 9, 2016
March 31, 2017
May 2016
April 2019   (Final data collection date for primary outcome measure)
  • Maximal lingual (tongue) strength measured via manual muscle testing (MMT) measured via ordinal scale (see description) [ Time Frame: Day 1 ]

    Lingual strength will be rated with a validated 0-4 ordinal scale.

    Score Description

    0 - Normal strength, no weakness.

    1. - Mild weakness. The tongue can be overcome with effort.
    2. - Moderate weakness. Easy to overcome.
    3. - Minimal movement. Unable to protrude to either side.
    4. - No movement detected.
  • Maximal lingual (tongue) strength measured via quantitative muscle testing (QMT) measured in kilopascals (KPA) [ Time Frame: Day 1 ]
Same as current
Complete list of historical versions of study NCT02765828 on ClinicalTrials.gov Archive Site
  • Maximal muscle thickness measured with ultrasound assessment in millimeters (mm) [ Time Frame: Day 1 ]
    Comprises part of assessment of lingual (tongue) structure via qualitative tongue ultrasound assessment. On-screen calipers will be used to perform measurement.
  • Echo intensity measured with ultrasound assessment utilizing grayscale analysis [ Time Frame: Day 1 ]
    Comprises part of assessment of lingual (tongue) structure via qualitative tongue ultrasound assessment. Echo intensity measurements consist of drawing a box over subcutaneous tissue and muscle areas of interest using the grayscale histogram function. This number will be recorded along with the standard deviation (grayscale analysis).
Same as current
Not Provided
Not Provided
 
Identification of Tongue Involvement in Late-Onset Pompe Disease
Determining the Diagnostic Utility of the Identification of Tongue Involvement in Late-Onset Pompe Disease (LOPD)
This purpose of this study is to determine if tongue strength and tongue ultrasound measurements differentiates patients with untreated late-onset Pompe Disease (LOPD) from patients with acquires/hereditary myopathies or neuropathies. It is hypothesized that abnormalities in tongue function and structure in patients with LOPD may be useful in discriminating this condition from others that have similar presentations.
Not Provided
Observational
Observational Model: Case-Control
Time Perspective: Cross-Sectional
Not Provided
Not Provided
Non-Probability Sample
Prospective participants must have a confirmed diagnosis of late-onset Pompe Disease, acquired/hereditary myopathy, or neuropathy.
  • Myopathy
  • Neuropathy
  • Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Other: Observational study
The following exams will be done in all cohorts: tongue manual muscle testing (MMT), tongue quantitative muscle testing, tongue ultrasound measurements
  • Late-Onset Pompe Disease
    Intervention: Other: Observational study
  • Acquired/Hereditary Myopathy
    Intervention: Other: Observational study
  • Neuropathy
    Intervention: Other: Observational study
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
70
April 2019
April 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • age ≥ 12 years
  • confirmed diagnosis of LOPD and naïve to enzyme-replacement therapy (ERT)
  • acquired/hereditary myopathy (e.g., dermatomyositis, polymyositis, inclusion body myositis, limb-girdle muscular dystrophy, distal myopathy, myotonic muscular dystrophy, and other myopathy)
  • neuropathy (e.g., peripheral neuropathy, cranial neuropathy, autonomic neuropathy, focal neuropathy)

Exclusion Criteria:

  • current use, history within the past two years of use, or eligible but declined use of Lumizyme® enzyme replacement therapy (applicable to LOPD group)
  • history of stroke, Parkinson's disease, oculopharyngeal muscular dystrophy, head and neck cancer or radiation treatment to head/neck, or other conditions that commonly affect lingual strength
  • inability to follow directions for study participation
Sexes Eligible for Study: All
12 Years and older   (Child, Adult, Senior)
No
Contact: Amy Walker amy.walker1@duke.edu
Contact: Kelly Crisp kelly.crisp@duke.edu
United States
 
 
NCT02765828
Pro00068729
No
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Duke University
Duke University
Genzyme, a Sanofi Company
Principal Investigator: Harrison Jones, PhD Division of Head and Neck Surgery & Communication Sciences, Duke University Medical Center
Duke University
March 2017