Identification of Tongue Involvement in Late-Onset Pompe Disease

Completed

Conditions studied: Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)

In brief

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.

Key facts

Study ID

NCT02765828

Run by

Duke University

People needed

73

Starts

2016-05-25

Expected to finish

2019-07-15

Last updated by the study team

2021-07-06

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Duke University Medical Center — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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