Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

Glycogen Storage Disease Clinical Trial

Official title:

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03255213
• Other study ID #: Pro00077344
• Status: Completed
• Phase: N/A
• Start date: March 21, 2018
• Est. completion date: April 23, 2020

Study information

• Verified date: July 2021
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: April 23, 2020
• Est. primary completion date: April 23, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II

Intervention

Other:
• Lingual Muscle Training
Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device

Locations

Country	Name	City	State
United States	Duke University Medical Center and affiliated practices	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Duke University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of lingual muscle training	Safety measured by pain rating scale	13 weeks
Primary	Feasibility of lingual muscle training as measured by questionnaires		13 weeks
Secondary	Change in lingual strength	Change from pre-test to post-test	baseline, 13 weeks