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

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.

Study Overview

• Status: Completed
• Conditions: Glycogen Storage Disease
• Intervention / Treatment: Other: Lingual Muscle Training

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center and affiliated practices

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Late Onset Pompe Disease who have tongue weakness	Other: Lingual Muscle Training; Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of lingual muscle training	Safety measured by pain rating scale	13 weeks
Feasibility of lingual muscle training as measured by questionnaires		13 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in lingual strength	Change from pre-test to post-test	baseline, 13 weeks

Collaborators and Investigators

• Sponsor: Duke University
• Investigators: Principal Investigator: Jones Harrison, PhD, Duke University

Study record dates

Study Major Dates

• Study Start (Actual): March 21, 2018
• Primary Completion (Actual): April 23, 2020
• Study Completion (Actual): April 23, 2020

Study Registration Dates

• First Submitted: August 3, 2017
• First Submitted That Met QC Criteria: August 17, 2017
• First Posted (Actual): August 21, 2017

Study Record Updates

• Last Update Posted (Actual): July 28, 2021
• Last Update Submitted That Met QC Criteria: July 27, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease Type II; Glycogen Storage Disease
• Other Study ID Numbers: Pro00077344

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No