Glycogen Storage Disease
Conditions
Brief summary
This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with Late-Onset Pompe Disease (LOPD) who have tongue weakness. The results of this study will help design future research studies about LMT in LOPD.
Interventions
OTHERLingual Muscle Training
Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device
Sponsors
Duke University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥ 12 years * Lingual weakness as measured by quantitative muscle testing (≤ 5% lower limit of normal for stratified age) * Confirmed diagnosis of LOPD * On enzyme replacement therapy for ≥ 26 weeks at pretest * Able to follow directions for study participation * Able to complete a home-based LMT regimen * Access to reliable internet connection * Access to use of electronic device that allows for FaceTime, Jabber, or WebEx use
Exclusion criteria
* Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator * Any current or past history of seizures. * Head and neck cancer or radiation treatment to head/neck * Inability to tolerate IOPI device tongue bulb * Inability to give legally effective consent * Inability to read and understand English
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of lingual muscle training | 13 weeks | Safety measured by pain rating scale |
| Feasibility of lingual muscle training as measured by questionnaires | 13 weeks | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in lingual strength | baseline, 13 weeks | Change from pre-test to post-test |
Countries
United States
Outcome results
None listed