Identification of Tongue Involvement in Late-Onset Pompe Disease

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Duke University

Status

Completed

Conditions

Myopathy
Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Neuropathy

Treatments

Other: Observational study

Study type

Observational

Funder types

Other
Industry

Identifiers

NCT02765828
Pro00068729

Details and patient eligibility

About

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.

Enrollment

73 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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

Trial design

73 participants in 3 patient groups

Late-Onset Pompe Disease
Treatment:
Other: Observational study
Acquired/Hereditary Myopathy
Treatment:
Other: Observational study
Neuropathy
Treatment:
Other: Observational study

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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