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

• Sponsor: Duke University (Other)
• Overall Status: Completed
• CT.gov ID: NCT03255213
• Collaborator: (none)
• Enrollment: 5
• Location: 1
• Arm: 1
• Actual Duration (Months): 25.1
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Glycogen Storage Disease	Other: Lingual Muscle Training	N/A

Study Design

• Study Type: Interventional
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
• Actual Study Start Date: Mar 21, 2018
• Actual Primary Completion Date: Apr 23, 2020
• Actual Study Completion Date: Apr 23, 2020

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Safety of lingual muscle training [13 weeks]

   Safety measured by pain rating scale

2. Feasibility of lingual muscle training as measured by questionnaires [13 weeks]

Secondary Outcome Measures

1. Change in lingual strength [baseline, 13 weeks]

   Change from pre-test to post-test

Eligibility Criteria

Criteria

• Ages Eligible for Study: 12 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Duke University Medical Center and affiliated practices	Durham	North Carolina	United States	27710

Sponsors and Collaborators

• Duke University

Investigators

• Principal Investigator: Jones Harrison, PhD, Duke University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Duke University
• ClinicalTrials.gov Identifier: NCT03255213
• Other Study ID Numbers: Pro00077344
• First Posted: Aug 21, 2017
• Last Update Posted: Jul 28, 2021
• Last Verified: Jul 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Glycogen Storage Disease Type II; Glycogen Storage Disease

Study Results

No Results Posted as of Jul 28, 2021