Identification of Tongue Involvement in Late-Onset Pompe Disease



Status:Recruiting
Conditions:Diabetes
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:12 - Any
Updated:3/31/2019
Start Date:May 2016
End Date:September 2019
Contact:Amy Walker
Email:amy.walker1@duke.edu

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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.


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
We found this trial at
1
site
2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Harrision Jones
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Durham, NC
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